Muscular Atrophy
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326 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Muscular Atrophy
ChiCTR-TRC-10001093: Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial

Completed
4
40
 
Rat nerve growth factor injection for six monthes, ;Rat nerve growth factor injection for six monthes,
PLA General Hospital; Xia Men Bioway Biotech Co., LTD, Xia Men Bioway Biotech Co., LTD
children with spinal muscular atrophy
 
 
NCT03648658 / 2018-002295-40: Paracetamol Study in Patients With Low Muscle Mass

Recruiting
4
48
Europe
Paracetamol 120Mg/5mL Oral Suspension
Mette Cathrine Oerngreen, Elsass Foundation
SMA II, Cerebral Palsy
12/24
12/24
2019-005007-40: Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen. Studie van het natuurlijk ziekteverloop bij volwassen patiënten met spinale spieratrofie (SMA) en de rol van biomerkers bij SMA patiënten die met nusinersen behandeld worden.

Not yet recruiting
4
19
Europe
Solution for injection, Spinraza
University Hospitals Leuven, Biogen
Adult patients with spinal muscular atrophy (SMA) type 2, type 3, or type 4, spinal muscular atrophy (SMA), Diseases [C] - Nervous System Diseases [C10]
 
 
ChiCTR2000041208: A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy

Recruiting
4
90
 
salbutamol sulfate tablets ;placebo
The Children's Hospital, Zhejiang University School of Medicine; Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd., Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd.
spinal muscular atrophy
 
 
OFELIA, NCT05073133: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Completed
4
16
RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Muscular Atrophy, Spinal
08/23
08/23
2023-000864-67: Safety and efficacy of intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)

Not yet recruiting
4
16
RoW
Solution for injection, Zolgensma
NOVARTIS BIOCIÊNCIAS S.A, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
RESPOND, NCT04488133 / 2020-003492-18: A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Active, not recruiting
4
46
Europe, US, RoW
Nusinersen, ISIS 396443, BIIB058, Spinraza
Biogen
Muscular Atrophy, Spinal
10/25
10/25
RISE, NCT05522361: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruiting
4
10
US
Risdiplam
Clinic for Special Children, Genentech, Inc.
Spinal Muscular Atrophy
12/25
06/26
ChiCTR2300072288: Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy (SMA): a Real-World, Multi-Center Cohort Study

Recruiting
4
60
 
NA
Peking University First Hospital; Peking University First Hospital, Shanghai Roche Pharmaceuticals Co., Ltd.
Spinal Muscular Atrophy (SMA)
 
 
HINALEA 1, NCT05861986: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting
4
28
Europe, US
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
HINALEA 2, NCT05861999: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

Recruiting
4
28
Europe, US, RoW
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
WeSMA, NCT05232929: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Active, not recruiting
4
402
US
Risdiplam, Evrysdi®, SMA-DAT Application and ADAM Sensor
Genentech, Inc.
Spinal Muscular Atrophy
12/26
12/26
ACTRN12619001372178: The effects of pubertal induction on bone health in children with neuromuscular conditions

Recruiting
3/4
58
 
Murdoch Childrens Research Institute, Foundation for Children
cerebral palsy, Spinal muscular atrophy , Muscular dystrophies
 
 
2021-006781-21: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials

Ongoing
3/4
260
Europe
OAV101, OAV101, Solution for infusion, Solution for injection, Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2020-005995-37: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) Sécurité et efficacité d'OAV101 (AVXS-101) administrée par voie intraveineuse, chez des patients pédiatriques atteints d’amyotrophie spinale (SMART)

Ongoing
3
30
Europe
OAV101, AVXS-101, Solution for infusion, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy Amyotrophie spinale, Spinal Muscular Atrophy Amyotrophie spinale, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT03179631: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Completed
3
360
Europe, Canada, Japan, US, RoW
Ataluren, PTC124, PLACEBO, Matching Placebo
PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
03/22
07/23
2021-006709-31: Phase IIIb, open-label, multi-center study to evaluate safety, tolerability and efficacy study of OAV101 administered intrathecally to participants with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen or risdiplam

Ongoing
3
28
Europe
OAV101, OAV101, Solution for injection, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
ChiCTR2400085770: Study on the effect of foam axis on the prevention of lower limb venous thrombosis and muscle atrophy in pregnant women with bed rest

Completed
3
110
 
Routine nursing measures to prevent deep venous thrombosis of lower limbs+ Foam axis independent exercise mode; Routine nursing measures to prevent deep venous thrombosis of lower limbs
Women's Hospital, School of Medicine, Zhejiang University; Women's Hospital, School of Medicine, Zhejiang University, Self-financing
Deep venous thrombosis; Muscle atrophy
 
 
2021-003474-31: Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)

Not yet recruiting
3
125
Europe
OAV101, Okrido, prednisolone, OAV101, Solution for injection, Oral solution, Zolgensma, Okrido 6mg/mL oral solution, Prednisolone Sodium Phosphate Oral Solution
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
SMART, NCT04851873: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Completed
3
24
Europe, Canada, US, RoW
OAV101
Novartis Pharmaceuticals
Spinal Muscular Atrophy
06/23
06/23
SHINE, NCT02594124 / 2015-001870-16: A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Completed
3
292
Europe, Canada, Japan, US, RoW
nusinersen, ISIS 396443, Spinraza, BIIB058, IONIS SMN Rx, ISIS SMNRx
Biogen
Spinal Muscular Atrophy
08/23
08/23
DEVOTE, NCT04089566 / 2019-002663-10: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Completed
3
145
Europe, Canada, Japan, US, RoW
Nusinersen, BIIB058
Biogen
Muscular Atrophy, Spinal
02/24
05/24
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Completed
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
12/24
12/24
STRENGTH, NCT05386680: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

Completed
3
27
Europe, Canada, Japan, US, RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy
11/24
11/24
HOPE-3, NCT05126758: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, not recruiting
3
104
US
Deramiocel (CAP-1002), Cardiosphere-Derived Cells (CDCs), Placebo
Capricor Inc.
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
12/25
12/27
STEER, NCT05089656: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Active, not recruiting
3
127
Europe, US, RoW
OAV101, Zolgensma, AVXS-101, Sham control
Novartis Pharmaceuticals
Type 2 Spinal Muscular Atrophy
11/24
04/25
RESILIENT, NCT05337553 / 2022-000193-25: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

Active, not recruiting
3
269
Europe, US, RoW
taldefgrobep alfa, BHV-2000, BMS-986089, Placebo
Biohaven Pharmaceuticals, Inc., Biohaven Pharmaceuticals, Inc, Biohaven Pharmaceuticals, Inc.
Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
01/25
01/25
ONWARD, NCT04729907 / 2020-004708-32: A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial

Active, not recruiting
3
115
Europe, Canada, Japan, US, RoW
Nusinersen, BIIB058, Spinraza
Biogen, Biogen Idec Research Limited
Muscular Atrophy, Spinal
07/26
07/26
ONYX, NCT05626855 / 2022-001771-14: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Active, not recruiting
3
238
Europe, US
Apitegromab
Scholar Rock, Inc., Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
11/26
05/29
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
NCT04042025 / 2019-002611-26: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active, not recruiting
3
85
Europe, Canada, Japan, US, RoW
Onasemnogene Abeparvovec-xioi, Zolgensma
Novartis Gene Therapies
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
12/35
12/35
NCT05335876: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Recruiting
3
175
Europe, Canada, Japan, US, RoW
onasemnogene abeparvovec, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
06/30
06/30
NCT04456530: Use of Testosterone to Prevent Post-Surgical Muscle Loss - Pilot Study

Enrolling by invitation
2/3
15
US
Aveed 750 MG in 3 ML IM Injection, Normal Saline 3 ML IM Injection
The Stone Research Foundation for Sports Medicine and Arthritis
Muscle Atrophy, Surgery, Knee Injuries and Disorders, Complication of Surgical Procedure, Trauma
12/21
06/22
Med-SBMA, NCT06862596: Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Recruiting
2/3
68
Japan
Mexiletine hydrochloride, Placebo
Masahisa Katsuno
Spinal and Bulbar Muscular Atrophy
12/27
12/27
MANATEE, NCT05115110 / 2021-003417-19: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

Active, not recruiting
2/3
259
Europe, Canada, Japan, US, RoW
RO7204239, Placebo, Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Spinal Muscular Atrophy (SMA)
02/29
02/29
ChiCTR-ONC-11001420: Study of effect of Umbilical cord Mesenchymal Stem Cell of Children with spinal muscular atrophy

Completed
2
10
 
stem cell transplantataion
General Hospital of Chinese people's Armed Police Forces; General Hospital of Chinese people's Armed Police Forces, General Hospital of Chinese people's Armed Police Forces
spinal muscular atrophy
 
 
2011-004369-34: SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA en Pyridostigmine in volwassenen en kinderen; experimentele trial naar effect van pyridostigmine vergeleken placebo in volwassen Nederlandse patienten met spinale spieratrofie type 2, 3 en 4.

Ongoing
2
45
Europe
Mestinon, RVG 03820, Tablet, pyridostigmine bromide
Universtiy Medical Center Utrecht, Prinses Beatrix Fonds
Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. Spinale spieratrofie (SMA) is een aandoening gekenmerkt door proximale spierzwakte door degeneratie van motorneuronen in de voorhoorncellen van het ruggenmerg. SMA wordt veroorzaakt door een homzygote deletie van het SMN1-gen. Er zijn 4 types SMA, geclassificeerd op basis van behaalde motorische mijlpalen en leeftijd van eerste symtpomen. De behandeling van SMA is ondersteunend., Spinal muscular atrophy (SMA) is a disorder characterized by muscle weakness caused by loss of motor cells of the spinal cord. SMA is a genentic disease. Treatment of SMA is supportive. Spinale spieratrofie (SMA) is een aandoening met proximale spierzwakte door verlies van motorische cellen in het ruggenmerg. SMA is een genetische aandoening. De behandeling van SMA is ondersteunend., Diseases [C] - Nervous System Diseases [C10]
 
 
2010-022558-18: A pilot trial with clenbuterol in spinal and bulbar muscular atrophy (SBMA). Studio pilota con il clenbuterolo nella atrofia muscolare spinale bulbare (SBMA)

Ongoing
2
15
Europe
MONORES*30CPR 20MCG, MONORES*30CPR 20MCG
AZIENDA OSPEDALIERA DI PADOVA
spinal and bulbar muscular atrophy (SBMA) atrofia muscolare spinale e bulbare (SBMA)
 
 
2018-000160-28: Clinical study evaluating the long term safety of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 Studio clinico che valuta la sicurezza alungo termine del farmaco amifampridina fosfato in pazienti deambulanti con Atrofia Muscolare Spinale (SMA) tipo 3

Not yet recruiting
2
12
Europe
Amifampridina fosfato, [Amifampridina fosfato], Tablet, FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
CATALYST PHARMACEUTICALS INC., Catalyst Pharmaceuticals, Inc.
Spinal Muscular Atrophy (SMA) Type 3 Atrofia Muscolare Spinale (SMA) tipo 3, Spinal Muscular Atrophy (SMA) Atrofia Muscolare Spinale (SMA), Diseases [C] - Nervous System Diseases [C10]
 
 
SUNFISH, NCT02908685 / 2016-000750-35: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Completed
2
231
Europe, Canada, Japan, US, RoW
Placebo, Risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
09/19
10/23
FIREFISH, NCT02913482 / 2016-000778-40: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Completed
2
62
Europe, Japan, US, RoW
Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
11/19
12/23
TOPAZ, NCT03897322 / 2018-004383-65: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Not yet recruiting
2
55
NA
SRK-015
Scholar Rock, Inc., Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations
01/21
04/21
TOPAZ, NCT03921528 / 2018-004383-65: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Completed
2
58
Europe, US
SRK-015
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations
01/21
02/24
2017-005103-27: A PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF CLENBUTEROL IN PATIENTS WITH SPINAL AND BULBAR MUSCULAR ATROPHY (SBMA) STUDIO DELL'EFFICACIA E DELLA SICUREZZA D'USO DEL CLENBUTEROLO NELLA MALATTIA DI KENNEDY

Not yet recruiting
2
90
Europe
MONORES 20 mcg 30 compresse, [024217034], Tablet, MONORES 20 mcg 30 compresse
AZIENDA OSPEDALIERA DI PADOVA, AIFA - Italian Medicines Agency
Motor neuron disease characterized by atrophy and muscle weakness in the spinal and bulbar region Malattia motoneuronale caratterizzata da atrofia e debolezza muscolare nel distretto spinale e bulbare, SBMA is an adult motor neuron disease that leads to muscle atrophy and weakness in the spinal and bulbar region. La SBMA è una malattia motoneuronale dell'età adulta che conduce a atrofia e debolezza muscolare nel distretto spinale e bulbare., Diseases [C] - Nervous System Diseases [C10]
 
 
NCT05398042: Ketone Administration During Inactivity and Retraining

Recruiting
2
24
Europe
Ketone ester, Medium Chain Triglyceride (MCT) oil
KU Leuven
Exercise, Healthy Male/Female Subjects
06/22
08/22
Rainbowfish, NCT03779334 / 2018-002087-12: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruiting
2
26
Europe, US, RoW
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
02/23
03/27
NCT05776862: Study Testing Benefits of Ursolic Acid (UA) as a Countermeasure To Myopenia and Insulin Resistance in Chronic Spinal Cord Injury (SCI)

Recruiting
2
20
US
Ursolic Acid, Strength Training
University of Miami, Florida
Spinal Cord Injuries, Tetraplegia, Paraplegia, Muscle Loss, Atrophy, Muscular, Insulin Resistance
06/25
06/25
NCT03332238: Stromal Vascular Fraction Cell Therapy to Improve the Repair of Rotator Cuff Tears

Active, not recruiting
2
56
US
Autologous Stomal Vascular Fraction Material, Ringer's solution
Hospital for Special Surgery, New York, Orthopedic Research and Education Foundation
Rotator Cuff Tear, Muscle Atrophy, Tendon Tear
06/25
07/25
PIONEER KD, NCT06411912: A Study of NIDO-361 in Patients With SBMA

Active, not recruiting
2
54
Europe, RoW
NIDO-361, Placebo
Nido Biosciences, Inc.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
10/25
10/25
BetaSBMA, NCT06169046: A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy

Recruiting
2
90
Europe
Clenbuterol, active, Placebo
Gianni Soraru, Mario Negri Institute for Pharmacological Research
Spinal and Bulbar Muscular Atrophy
06/27
12/27
ZABAS, NCT04742010: Zoledronic Acid for Prevention of Bone Loss After BAriatric Surgery

Recruiting
2
60
Europe
Zoledronic Acid, Placebo
Stinus Gadegaard Hansen, Research Unit of Health Sciences, Hospital of South West Jutland, Department of Regional Health Research, University of Southern Denmark, OPEN - Odense Patient data Explorative Network, The University of Southern Denmark, Odense, Denmark, Department of Radiology and Nuclear Medicine, Hospital of Southwest Jutland, 6700 Esbjerg, Denmark, Odense University Hospital
Bone Loss, Muscle Atrophy, Bariatric Surgery
06/24
06/24
FORTITUDE-OLE, NCT06547216: Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Enrolling by invitation
2
84
Europe, Canada, US
AOC 1020, delpacibart braxlosiran, del-brax
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
04/28
04/28
NCT05174611: Vitamin D to Improve Quadricep Muscle Strength

Recruiting
2
60
RoW
Vitamin D3, Placebo
Chinese University of Hong Kong
Anterior Cruciate Ligament Injuries, Quadriceps Muscle Atrophy, Anterior Cruciate Ligament Rupture, Anterior Cruciate Ligament Tear
03/28
09/29
SYNAPSE-SMA, NCT05794139 / 2022-002301-24: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Recruiting
2
54
Europe, Canada, US
NMD670, Placebo
NMD Pharma A/S, NMD Pharma A/S
Spinal Muscular Atrophy
01/26
01/26
NCT06050668: Essential Amino Acid Supplementation for Femoral Fragility Fractures

Recruiting
2
60
US
Essential Amino Acid Supplementation, MEND Repair & Recover
Michael C Willey, MEND, Slocum Research & Education Foundation
Femoral Fracture, Fragility Fracture, Muscle Atrophy
07/25
12/25
Jewelfish, NCT03032172 / 2016-004184-39: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Completed
2
174
Europe, US
Risdiplam, RO7034067
Hoffmann-La Roche
Spinal Muscular Atrophy
02/25
02/25
NURTURE, NCT02386553 / 2014-002098-12: A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Completed
2
25
Europe, Canada, US, RoW
Nusinersen, ISIS 396443, BIIB058, ISIS SMNRx, Spinraza
Biogen
Spinal Muscular Atrophy
12/24
12/24
MARINA-OLE, NCT05479981: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Active, not recruiting
2
37
US
Placebo, Saline, AOC 1001
Avidity Biosciences, Inc.
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
06/27
06/27
Pupfish, NCT05808764: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Recruiting
2
10
Europe, Canada, US
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
10/25
10/25
NCT03150511: Tesamorelin to Improve Functional Outcomes After Peripheral Nerve Injury

Recruiting
2
36
US
Tesamorelin 2 Milligrams (MG), Egrifta
Johns Hopkins University, United States Department of Defense
Peripheral Nerve Injuries
12/25
12/25
NCT02268552 / 2014-002053-19: An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Completed
1/2
40
Europe, RoW
branaplam
Novartis Pharmaceuticals
Spinal Muscular Atrophy
12/22
12/22
NCT05211986: Safety and Tolerability of IMM01-STEM in Patients With Muscle Atrophy Related to Knee Osteoarthritis.

Completed
1/2
13
US
IMM01-STEM
Immunis, Inc.
Muscle Atrophy
08/24
11/24
ChiCTR2200056833: Single arm and single center clinical study on safety, tolerance and efficacy of GC101 injection in patients with spinal muscular atrophy type 1

Completed
1/2
3
 
Intrathecal injection of GC101
Seventh Medical Center of PLA General Hospital; Seventh Medical Center of PLA General Hospital, E-Town cooperation & Development Foundation
type 1 spinal muscular atrophy (SMA1)
 
 
NCT05517603: A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Completed
1/2
25
US
AJ201, JM17, Placebo
AnnJi Pharmaceutical Co., Ltd.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
04/24
04/24
NCT06421831: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Recruiting
1/2
21
RoW
GC101
GeneCradle Inc
Spinal Muscular Atrophy Type 3
12/26
12/28
NCT06288230: An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

Recruiting
1/2
6
RoW
vesemnogene lantuparvovec, AAV-hSMN1
Lantu Biopharma
Spinal Muscular Atrophy
10/27
10/27
NCT05614531: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Enrolling by invitation
1/2
12
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy Type I
08/25
08/25
NCT05901987: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Recruiting
1/2
33
RoW
GC101
GeneCradle Inc
SMA II
05/25
12/28
BLUEBELL, NCT05747261: Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy

Recruiting
1/2
40
RoW
ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
Biocad
Spinal Muscular Atrophy (SMA)
09/25
08/30
FORTITUDE, NCT05747924: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Active, not recruiting
1/2
90
Europe, Canada, US
AOC 1020, Placebo, Saline
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
10/26
04/27
NCT05824169: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Recruiting
1/2
18
RoW
GC101
GeneCradle Inc
Spinal Muscular Atrophy
12/25
12/25
ChiCTR2200066399: Multi center, open, single arm, single dose increasing phase I/IIa clinical trial to evaluate the safety, tolerance and initial efficacy of GC101 adeno-associated virus injection in the treatment of type 1 SMA patients

Not yet recruiting
1/2
15
 
GC101 intrathecal injection
The seventh medical center of PLA General Hospital; Beijing Jinlan Gene Technology Co., Ltd, raise independently
type 1 spinal muscular atrophy
 
 
2022-000956-12: BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma

Not yet recruiting
1
24
Europe
BIIB115, Solution for injection
Biogen Idec Research Limited, Biogen Idec Research Limited
Spinal muscular atrophy, SMA, Diseases [C] - Nervous System Diseases [C10]
 
 
2006-006845-14: Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.

Ongoing
1
20
Europe
TRO19622,
TROPHOS
Spinal Muscular Atrophy (SMA)
 
 
ChiCTR1800018378: A clinical trial and application of pulsed radiofrequency treatment of genicular nerves for muscle atrophy in knee osteoarthritis

Not yet recruiting
1
60
 
pulsed radiofrequency treatment ;Knee pain points lidocaine and diprospan injection
Huadong Hospital Affiliated To Fudan University; Level of the institution：, raise independently
Knee Osteoarthritis
 
 
ChiCTR-IIR-17013983: Effect of Xiaoyu Xiezhuo Yin on Muscular Atrophy Induced by Chronic Renal Failure through IGF-1 / PI3K / Akt Signaling Pathway

Not yet recruiting
1
100
 
Based on Western medicine treatment, add Xiaoyu Xiezhuo drink. ;Only Western medicine treatment
Zhejiang Provincial Hospital of TCM; Zhejiang Provincial Hospital of TCM, Zhejiang Provincial Hospital of TCM
chronic kidney disease
 
 
EXG001-307-101, NCT06576388: Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

Completed
1
2
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Type I Spinal Muscular Atrophy
09/23
01/24
NCT04708847: A Clinical Pharmacology Study to Evaluate the Effect of GYM329 on Disuse Muscle Atrophy in Healthy Volunteer

Completed
1
30
Europe
GYM329, Placebo
Chugai Pharmaceutical
Healthy Volunteers
06/22
02/23
NCT04718181: Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

Completed
1
131
US
risdiplam, Evrysdi, omeprazole, Non-investigational medicinal product
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/23
01/23
ACTRN12622000964718: A Phase 1 trial assessing the Safety, Tolerability, and Pharmacokinetics of NIDO-361 in Healthy Male Subjects

Completed
1
64
 
Nido Biosciences, Inc., Nido Biosciences, Inc.
Spinal and bulbar muscular atrophy (SBMA)
 
 
NCT05730842: Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers

Completed
1
15
US
EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
Edgewise Therapeutics, Inc.
Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn
04/23
04/23
ChiCTR2200064801: The effect mechanism of exercise through the intestinal - muscle axis to intervene the aging muscle atrophy

Not yet recruiting
1
400
 
exercise training ;none
Beijing Sport University; Beijing Sport University, Special funds from the central government
sarcopenia
 
 
ChiCTR2200063230: Single arm, single center, high-dose clinical study on the safety, tolerance and efficacy of GC101 adeno-associated virus injection in patients with type 1 spinal muscular atrophy

Recruiting
1
4
 
GC101 intrathecal injection
The Seventh Medical Center of PLA General Hospital ; The Seventh Medical Center of PLA General Hospital, fund projects
type 1 spinal muscular atrophy
 
 
NCT04057833: E-CEL UVEC Cells as an Adjunct Cell Therapy for the Arthroscopic Rotator Cuff Repair in Adults

Active, not recruiting
1
20
US
E-CEL UVEC
Hospital for Special Surgery, New York, Angiocrine Bioscience
Rotator Cuff Tears, Muscle Atrophy or Weakness, Tendon Rupture - Shoulder, Safety Issues
10/24
10/24
ChiCTR2200063672: EC50 of propofol with target controlled infusion for intrathecal injection of Nusinersen in children with spinal muscular atrophy

Not yet recruiting
1
30
 
Target controlled infusion of propofol for intravenous anesthesia
The First Affiliated Hospital of Fujian Medical University; The First Affiliated Hospital of Fujian Medical University, self-raised
Spinal muscular atrophy
 
 
ChiCTR2100054441: Single arm and single center clinical study on safety, tolerance and efficacy of GC101 injection in patients with type 2 and 3 spinal muscular atrophy

Completed
1
10
 
GC101 intrathecal injection
Seventh Medical Center of PLA General Hospital; Seventh Medical Center of PLA General Hospital, Fund projects
type 2 and 3 spinal muscular atrophy
 
 
NCT03107884: Role of Metformin on Muscle Health of Older Adults

Active, not recruiting
1
64
US
Metformin (Bed Rest), Metformin hydrochloride, Fortamet, glucophage, Glumetza, Riomet, Placebo (Bed Rest), Metformin (2 week run-in only), Glucophage, Placebo (2 week run-in only)
University of Utah
Muscle Atrophy, Insulin Resistance
11/25
11/25
NCT06185179: Metformin and Muscle Recovery

Recruiting
1
50
US
Metformin, Glucophage, Fortamet, Glumetza, Riomet, Placebo
University of Utah
Muscle Atrophy or Weakness
05/29
05/30
PIERRE-PK, NCT06555419: A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy

Recruiting
1
55
US
Nusinersen, BIIB058, ThecaFlex DRx System
Biogen
Muscular Atrophy, Spinal
12/25
12/25
NCT06888661: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy

Enrolling by invitation
1
4
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy (SMA)
03/26
12/26
NCT05575011 / 2022-000956-12: A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

Recruiting
1
62
Europe, Canada, RoW
BIIB115, BIIB115-Matching Placebo
Biogen, Biogen Idec Research Limited
Healthy Volunteer, Muscular Atrophy, Spinal
09/27
09/27
ChiCTR2500095667: An open label, single arm IIT clinical study evaluating the safety, tolerability, and preliminary efficacy of GCB-001 in the treatment of patients with delayed onset type 2 SMA who can sit alone but cannot walk

Not yet recruiting
1
6
 
Intrathecal injection Low Dosing GCB-001; Intrathecal injection High Dosing GCB-001
The Children's Hospital of Zhejiang University School of Medicine; The Children's Hospital of Zhejiang University School of Medicine, Enterprise funding
Spinal muscular atrophy
 
 
ACTRN12623000265673: Foot muscle measures of strength and cross-sectional area by ultrasound in the diabetic population.

Not yet recruiting
N/A
21
 
The University of Sydney , N/A
Diabetic peripheral neuropathy , Diabetic foot changes , Intrinsic foot muscle atrophy , Intrinsic foot muscle weakness
 
 
NCT06877689: EAP of Apitegromab for Patients With Spinal Muscular Atrophy

Available
N/A
NA
Apitegromab, SRK-015
Scholar Rock, Inc.
SMA
 
 
NCT00443066: Clinical Study of Spinal Muscular Atrophy

Completed
N/A
120
US
Columbia University, The Spinal Muscular Atrophy Foundation
Spinal Muscular Atrophy
08/11
08/11
ChiCTR-IPR-15007273: The effect of nutrition support combining with early rehabilitation on muscle mass and strength in ICU patients

Recruiting
N/A
60
 
early rehabilitation ;routine treatment
Beijing Tongren Hospital, Capital medical university; Beijing Tongren Hospital, Capital medical university, Self-raised funds
critical illness muscle atrophy
 
 
NCT06666816: Observational Study to Observe Variations of Gait Parameters in Patients with Neuromuscular Diseases

Recruiting
N/A
120
Europe
inapplicable
IRCCS Eugenio Medea
Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
12/26
12/26
SMA, NCT03217578: Neonatal Spinal Muscular Atrophy () Screening

Recruiting
N/A
250000
RoW
SMA Newborn Screening
Kaohsiung Medical University Chung-Ho Memorial Hospital, The Chinese Foundation of Health, Taipei Institute of Pathology
Spinal Muscular Atrophy
12/30
12/30
ChiCTR1800019722: Application of MUNIX in evaluating the surgical effect of patients with cervical spondylosis muscular atrophy

Recruiting
N/A
70
 
operation ;operation ;non-operation
Huashan Hospital, Fudan University; Huashan Hospital, Fudan University, Shanghai City Health System of the Second Batch of Important Diseases Combined Project (2014ZYJ0008)
Cervical Spondylotic Amyotrophy
 
 
ChiCTR1900026896: Effect of acupuncture regulating macrophage polarization to alleviate sepsis-induced muscle atrophy: a prospective study

Suspended
N/A
98
 
acupuncture+basic treatment ;basic treatment
Department of Intensive Care Unit, the First Affiliated Hospital of Guangzhou University of Chinese Medicine; The First Affiliated Hospital of Guangzhou University of Chinese Medicine, Fund of Guangdong Provincial Traditional Chinese Medicine Bureau Research Project, China (20192018), Foundation of Innovative Development Project (2017QN05)
Sepsis-induced myopathy
 
 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Muscular Atrophy
ChiCTR-TRC-10001093: Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial

Completed
4
40
 
Rat nerve growth factor injection for six monthes, ;Rat nerve growth factor injection for six monthes,
PLA General Hospital; Xia Men Bioway Biotech Co., LTD, Xia Men Bioway Biotech Co., LTD
children with spinal muscular atrophy
 
 
NCT03648658 / 2018-002295-40: Paracetamol Study in Patients With Low Muscle Mass

Recruiting
4
48
Europe
Paracetamol 120Mg/5mL Oral Suspension
Mette Cathrine Oerngreen, Elsass Foundation
SMA II, Cerebral Palsy
12/24
12/24
2019-005007-40: Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen. Studie van het natuurlijk ziekteverloop bij volwassen patiënten met spinale spieratrofie (SMA) en de rol van biomerkers bij SMA patiënten die met nusinersen behandeld worden.

Not yet recruiting
4
19
Europe
Solution for injection, Spinraza
University Hospitals Leuven, Biogen
Adult patients with spinal muscular atrophy (SMA) type 2, type 3, or type 4, spinal muscular atrophy (SMA), Diseases [C] - Nervous System Diseases [C10]
 
 
ChiCTR2000041208: A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy

Recruiting
4
90
 
salbutamol sulfate tablets ;placebo
The Children's Hospital, Zhejiang University School of Medicine; Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd., Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd.
spinal muscular atrophy
 
 
OFELIA, NCT05073133: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Completed
4
16
RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Muscular Atrophy, Spinal
08/23
08/23
2023-000864-67: Safety and efficacy of intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)

Not yet recruiting
4
16
RoW
Solution for injection, Zolgensma
NOVARTIS BIOCIÊNCIAS S.A, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
RESPOND, NCT04488133 / 2020-003492-18: A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Active, not recruiting
4
46
Europe, US, RoW
Nusinersen, ISIS 396443, BIIB058, Spinraza
Biogen
Muscular Atrophy, Spinal
10/25
10/25
RISE, NCT05522361: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruiting
4
10
US
Risdiplam
Clinic for Special Children, Genentech, Inc.
Spinal Muscular Atrophy
12/25
06/26
ChiCTR2300072288: Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy (SMA): a Real-World, Multi-Center Cohort Study

Recruiting
4
60
 
NA
Peking University First Hospital; Peking University First Hospital, Shanghai Roche Pharmaceuticals Co., Ltd.
Spinal Muscular Atrophy (SMA)
 
 
HINALEA 1, NCT05861986: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting
4
28
Europe, US
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
HINALEA 2, NCT05861999: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

Recruiting
4
28
Europe, US, RoW
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
WeSMA, NCT05232929: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Active, not recruiting
4
402
US
Risdiplam, Evrysdi®, SMA-DAT Application and ADAM Sensor
Genentech, Inc.
Spinal Muscular Atrophy
12/26
12/26
ACTRN12619001372178: The effects of pubertal induction on bone health in children with neuromuscular conditions

Recruiting
3/4
58
 
Murdoch Childrens Research Institute, Foundation for Children
cerebral palsy, Spinal muscular atrophy , Muscular dystrophies
 
 
2021-006781-21: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials

Ongoing
3/4
260
Europe
OAV101, OAV101, Solution for infusion, Solution for injection, Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2020-005995-37: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) Sécurité et efficacité d'OAV101 (AVXS-101) administrée par voie intraveineuse, chez des patients pédiatriques atteints d’amyotrophie spinale (SMART)

Ongoing
3
30
Europe
OAV101, AVXS-101, Solution for infusion, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy Amyotrophie spinale, Spinal Muscular Atrophy Amyotrophie spinale, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT03179631: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Completed
3
360
Europe, Canada, Japan, US, RoW
Ataluren, PTC124, PLACEBO, Matching Placebo
PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
03/22
07/23
2021-006709-31: Phase IIIb, open-label, multi-center study to evaluate safety, tolerability and efficacy study of OAV101 administered intrathecally to participants with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen or risdiplam

Ongoing
3
28
Europe
OAV101, OAV101, Solution for injection, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
ChiCTR2400085770: Study on the effect of foam axis on the prevention of lower limb venous thrombosis and muscle atrophy in pregnant women with bed rest

Completed
3
110
 
Routine nursing measures to prevent deep venous thrombosis of lower limbs+ Foam axis independent exercise mode; Routine nursing measures to prevent deep venous thrombosis of lower limbs
Women's Hospital, School of Medicine, Zhejiang University; Women's Hospital, School of Medicine, Zhejiang University, Self-financing
Deep venous thrombosis; Muscle atrophy
 
 
2021-003474-31: Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)

Not yet recruiting
3
125
Europe
OAV101, Okrido, prednisolone, OAV101, Solution for injection, Oral solution, Zolgensma, Okrido 6mg/mL oral solution, Prednisolone Sodium Phosphate Oral Solution
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
SMART, NCT04851873: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Completed
3
24
Europe, Canada, US, RoW
OAV101
Novartis Pharmaceuticals
Spinal Muscular Atrophy
06/23
06/23
SHINE, NCT02594124 / 2015-001870-16: A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Completed
3
292
Europe, Canada, Japan, US, RoW
nusinersen, ISIS 396443, Spinraza, BIIB058, IONIS SMN Rx, ISIS SMNRx
Biogen
Spinal Muscular Atrophy
08/23
08/23
DEVOTE, NCT04089566 / 2019-002663-10: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Completed
3
145
Europe, Canada, Japan, US, RoW
Nusinersen, BIIB058
Biogen
Muscular Atrophy, Spinal
02/24
05/24
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Completed
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
12/24
12/24
STRENGTH, NCT05386680: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

Completed
3
27
Europe, Canada, Japan, US, RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy
11/24
11/24
HOPE-3, NCT05126758: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, not recruiting
3
104
US
Deramiocel (CAP-1002), Cardiosphere-Derived Cells (CDCs), Placebo
Capricor Inc.
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
12/25
12/27
STEER, NCT05089656: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Active, not recruiting
3
127
Europe, US, RoW
OAV101, Zolgensma, AVXS-101, Sham control
Novartis Pharmaceuticals
Type 2 Spinal Muscular Atrophy
11/24
04/25
RESILIENT, NCT05337553 / 2022-000193-25: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

Active, not recruiting
3
269
Europe, US, RoW
taldefgrobep alfa, BHV-2000, BMS-986089, Placebo
Biohaven Pharmaceuticals, Inc., Biohaven Pharmaceuticals, Inc, Biohaven Pharmaceuticals, Inc.
Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
01/25
01/25
ONWARD, NCT04729907 / 2020-004708-32: A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial

Active, not recruiting
3
115
Europe, Canada, Japan, US, RoW
Nusinersen, BIIB058, Spinraza
Biogen, Biogen Idec Research Limited
Muscular Atrophy, Spinal
07/26
07/26
ONYX, NCT05626855 / 2022-001771-14: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Active, not recruiting
3
238
Europe, US
Apitegromab
Scholar Rock, Inc., Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
11/26
05/29
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
NCT04042025 / 2019-002611-26: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active, not recruiting
3
85
Europe, Canada, Japan, US, RoW
Onasemnogene Abeparvovec-xioi, Zolgensma
Novartis Gene Therapies
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
12/35
12/35
NCT05335876: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Recruiting
3
175
Europe, Canada, Japan, US, RoW
onasemnogene abeparvovec, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
06/30
06/30
NCT04456530: Use of Testosterone to Prevent Post-Surgical Muscle Loss - Pilot Study

Enrolling by invitation
2/3
15
US
Aveed 750 MG in 3 ML IM Injection, Normal Saline 3 ML IM Injection
The Stone Research Foundation for Sports Medicine and Arthritis
Muscle Atrophy, Surgery, Knee Injuries and Disorders, Complication of Surgical Procedure, Trauma
12/21
06/22
Med-SBMA, NCT06862596: Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Recruiting
2/3
68
Japan
Mexiletine hydrochloride, Placebo
Masahisa Katsuno
Spinal and Bulbar Muscular Atrophy
12/27
12/27
MANATEE, NCT05115110 / 2021-003417-19: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

Active, not recruiting
2/3
259
Europe, Canada, Japan, US, RoW
RO7204239, Placebo, Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Spinal Muscular Atrophy (SMA)
02/29
02/29
ChiCTR-ONC-11001420: Study of effect of Umbilical cord Mesenchymal Stem Cell of Children with spinal muscular atrophy

Completed
2
10
 
stem cell transplantataion
General Hospital of Chinese people's Armed Police Forces; General Hospital of Chinese people's Armed Police Forces, General Hospital of Chinese people's Armed Police Forces
spinal muscular atrophy
 
 
2011-004369-34: SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA en Pyridostigmine in volwassenen en kinderen; experimentele trial naar effect van pyridostigmine vergeleken placebo in volwassen Nederlandse patienten met spinale spieratrofie type 2, 3 en 4.

Ongoing
2
45
Europe
Mestinon, RVG 03820, Tablet, pyridostigmine bromide
Universtiy Medical Center Utrecht, Prinses Beatrix Fonds
Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. Spinale spieratrofie (SMA) is een aandoening gekenmerkt door proximale spierzwakte door degeneratie van motorneuronen in de voorhoorncellen van het ruggenmerg. SMA wordt veroorzaakt door een homzygote deletie van het SMN1-gen. Er zijn 4 types SMA, geclassificeerd op basis van behaalde motorische mijlpalen en leeftijd van eerste symtpomen. De behandeling van SMA is ondersteunend., Spinal muscular atrophy (SMA) is a disorder characterized by muscle weakness caused by loss of motor cells of the spinal cord. SMA is a genentic disease. Treatment of SMA is supportive. Spinale spieratrofie (SMA) is een aandoening met proximale spierzwakte door verlies van motorische cellen in het ruggenmerg. SMA is een genetische aandoening. De behandeling van SMA is ondersteunend., Diseases [C] - Nervous System Diseases [C10]
 
 
2010-022558-18: A pilot trial with clenbuterol in spinal and bulbar muscular atrophy (SBMA). Studio pilota con il clenbuterolo nella atrofia muscolare spinale bulbare (SBMA)

Ongoing
2
15
Europe
MONORES*30CPR 20MCG, MONORES*30CPR 20MCG
AZIENDA OSPEDALIERA DI PADOVA
spinal and bulbar muscular atrophy (SBMA) atrofia muscolare spinale e bulbare (SBMA)
 
 
2018-000160-28: Clinical study evaluating the long term safety of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 Studio clinico che valuta la sicurezza alungo termine del farmaco amifampridina fosfato in pazienti deambulanti con Atrofia Muscolare Spinale (SMA) tipo 3

Not yet recruiting
2
12
Europe
Amifampridina fosfato, [Amifampridina fosfato], Tablet, FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
CATALYST PHARMACEUTICALS INC., Catalyst Pharmaceuticals, Inc.
Spinal Muscular Atrophy (SMA) Type 3 Atrofia Muscolare Spinale (SMA) tipo 3, Spinal Muscular Atrophy (SMA) Atrofia Muscolare Spinale (SMA), Diseases [C] - Nervous System Diseases [C10]
 
 
SUNFISH, NCT02908685 / 2016-000750-35: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Completed
2
231
Europe, Canada, Japan, US, RoW
Placebo, Risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
09/19
10/23
FIREFISH, NCT02913482 / 2016-000778-40: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Completed
2
62
Europe, Japan, US, RoW
Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
11/19
12/23
TOPAZ, NCT03897322 / 2018-004383-65: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Not yet recruiting
2
55
NA
SRK-015
Scholar Rock, Inc., Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations
01/21
04/21
TOPAZ, NCT03921528 / 2018-004383-65: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Completed
2
58
Europe, US
SRK-015
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations
01/21
02/24
2017-005103-27: A PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF CLENBUTEROL IN PATIENTS WITH SPINAL AND BULBAR MUSCULAR ATROPHY (SBMA) STUDIO DELL'EFFICACIA E DELLA SICUREZZA D'USO DEL CLENBUTEROLO NELLA MALATTIA DI KENNEDY

Not yet recruiting
2
90
Europe
MONORES 20 mcg 30 compresse, [024217034], Tablet, MONORES 20 mcg 30 compresse
AZIENDA OSPEDALIERA DI PADOVA, AIFA - Italian Medicines Agency
Motor neuron disease characterized by atrophy and muscle weakness in the spinal and bulbar region Malattia motoneuronale caratterizzata da atrofia e debolezza muscolare nel distretto spinale e bulbare, SBMA is an adult motor neuron disease that leads to muscle atrophy and weakness in the spinal and bulbar region. La SBMA è una malattia motoneuronale dell'età adulta che conduce a atrofia e debolezza muscolare nel distretto spinale e bulbare., Diseases [C] - Nervous System Diseases [C10]
 
 
NCT05398042: Ketone Administration During Inactivity and Retraining

Recruiting
2
24
Europe
Ketone ester, Medium Chain Triglyceride (MCT) oil
KU Leuven
Exercise, Healthy Male/Female Subjects
06/22
08/22
Rainbowfish, NCT03779334 / 2018-002087-12: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruiting
2
26
Europe, US, RoW
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
02/23
03/27
NCT05776862: Study Testing Benefits of Ursolic Acid (UA) as a Countermeasure To Myopenia and Insulin Resistance in Chronic Spinal Cord Injury (SCI)

Recruiting
2
20
US
Ursolic Acid, Strength Training
University of Miami, Florida
Spinal Cord Injuries, Tetraplegia, Paraplegia, Muscle Loss, Atrophy, Muscular, Insulin Resistance
06/25
06/25
NCT03332238: Stromal Vascular Fraction Cell Therapy to Improve the Repair of Rotator Cuff Tears

Active, not recruiting
2
56
US
Autologous Stomal Vascular Fraction Material, Ringer's solution
Hospital for Special Surgery, New York, Orthopedic Research and Education Foundation
Rotator Cuff Tear, Muscle Atrophy, Tendon Tear
06/25
07/25
PIONEER KD, NCT06411912: A Study of NIDO-361 in Patients With SBMA

Active, not recruiting
2
54
Europe, RoW
NIDO-361, Placebo
Nido Biosciences, Inc.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
10/25
10/25
BetaSBMA, NCT06169046: A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy

Recruiting
2
90
Europe
Clenbuterol, active, Placebo
Gianni Soraru, Mario Negri Institute for Pharmacological Research
Spinal and Bulbar Muscular Atrophy
06/27
12/27
ZABAS, NCT04742010: Zoledronic Acid for Prevention of Bone Loss After BAriatric Surgery

Recruiting
2
60
Europe
Zoledronic Acid, Placebo
Stinus Gadegaard Hansen, Research Unit of Health Sciences, Hospital of South West Jutland, Department of Regional Health Research, University of Southern Denmark, OPEN - Odense Patient data Explorative Network, The University of Southern Denmark, Odense, Denmark, Department of Radiology and Nuclear Medicine, Hospital of Southwest Jutland, 6700 Esbjerg, Denmark, Odense University Hospital
Bone Loss, Muscle Atrophy, Bariatric Surgery
06/24
06/24
FORTITUDE-OLE, NCT06547216: Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Enrolling by invitation
2
84
Europe, Canada, US
AOC 1020, delpacibart braxlosiran, del-brax
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
04/28
04/28
NCT05174611: Vitamin D to Improve Quadricep Muscle Strength

Recruiting
2
60
RoW
Vitamin D3, Placebo
Chinese University of Hong Kong
Anterior Cruciate Ligament Injuries, Quadriceps Muscle Atrophy, Anterior Cruciate Ligament Rupture, Anterior Cruciate Ligament Tear
03/28
09/29
SYNAPSE-SMA, NCT05794139 / 2022-002301-24: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Recruiting
2
54
Europe, Canada, US
NMD670, Placebo
NMD Pharma A/S, NMD Pharma A/S
Spinal Muscular Atrophy
01/26
01/26
NCT06050668: Essential Amino Acid Supplementation for Femoral Fragility Fractures

Recruiting
2
60
US
Essential Amino Acid Supplementation, MEND Repair & Recover
Michael C Willey, MEND, Slocum Research & Education Foundation
Femoral Fracture, Fragility Fracture, Muscle Atrophy
07/25
12/25
Jewelfish, NCT03032172 / 2016-004184-39: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Completed
2
174
Europe, US
Risdiplam, RO7034067
Hoffmann-La Roche
Spinal Muscular Atrophy
02/25
02/25
NURTURE, NCT02386553 / 2014-002098-12: A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Completed
2
25
Europe, Canada, US, RoW
Nusinersen, ISIS 396443, BIIB058, ISIS SMNRx, Spinraza
Biogen
Spinal Muscular Atrophy
12/24
12/24
MARINA-OLE, NCT05479981: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Active, not recruiting
2
37
US
Placebo, Saline, AOC 1001
Avidity Biosciences, Inc.
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
06/27
06/27
Pupfish, NCT05808764: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Recruiting
2
10
Europe, Canada, US
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
10/25
10/25
NCT03150511: Tesamorelin to Improve Functional Outcomes After Peripheral Nerve Injury

Recruiting
2
36
US
Tesamorelin 2 Milligrams (MG), Egrifta
Johns Hopkins University, United States Department of Defense
Peripheral Nerve Injuries
12/25
12/25
NCT02268552 / 2014-002053-19: An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Completed
1/2
40
Europe, RoW
branaplam
Novartis Pharmaceuticals
Spinal Muscular Atrophy
12/22
12/22
NCT05211986: Safety and Tolerability of IMM01-STEM in Patients With Muscle Atrophy Related to Knee Osteoarthritis.

Completed
1/2
13
US
IMM01-STEM
Immunis, Inc.
Muscle Atrophy
08/24
11/24
ChiCTR2200056833: Single arm and single center clinical study on safety, tolerance and efficacy of GC101 injection in patients with spinal muscular atrophy type 1

Completed
1/2
3
 
Intrathecal injection of GC101
Seventh Medical Center of PLA General Hospital; Seventh Medical Center of PLA General Hospital, E-Town cooperation & Development Foundation
type 1 spinal muscular atrophy (SMA1)
 
 
NCT05517603: A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Completed
1/2
25
US
AJ201, JM17, Placebo
AnnJi Pharmaceutical Co., Ltd.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
04/24
04/24
NCT06421831: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Recruiting
1/2
21
RoW
GC101
GeneCradle Inc
Spinal Muscular Atrophy Type 3
12/26
12/28
NCT06288230: An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

Recruiting
1/2
6
RoW
vesemnogene lantuparvovec, AAV-hSMN1
Lantu Biopharma
Spinal Muscular Atrophy
10/27
10/27
NCT05614531: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Enrolling by invitation
1/2
12
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy Type I
08/25
08/25
NCT05901987: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Recruiting
1/2
33
RoW
GC101
GeneCradle Inc
SMA II
05/25
12/28
BLUEBELL, NCT05747261: Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy

Recruiting
1/2
40
RoW
ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
Biocad
Spinal Muscular Atrophy (SMA)
09/25
08/30
FORTITUDE, NCT05747924: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Active, not recruiting
1/2
90
Europe, Canada, US
AOC 1020, Placebo, Saline
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
10/26
04/27
NCT05824169: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Recruiting
1/2
18
RoW
GC101
GeneCradle Inc
Spinal Muscular Atrophy
12/25
12/25
ChiCTR2200066399: Multi center, open, single arm, single dose increasing phase I/IIa clinical trial to evaluate the safety, tolerance and initial efficacy of GC101 adeno-associated virus injection in the treatment of type 1 SMA patients

Not yet recruiting
1/2
15
 
GC101 intrathecal injection
The seventh medical center of PLA General Hospital; Beijing Jinlan Gene Technology Co., Ltd, raise independently
type 1 spinal muscular atrophy
 
 
2022-000956-12: BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma

Not yet recruiting
1
24
Europe
BIIB115, Solution for injection
Biogen Idec Research Limited, Biogen Idec Research Limited
Spinal muscular atrophy, SMA, Diseases [C] - Nervous System Diseases [C10]
 
 
2006-006845-14: Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.

Ongoing
1
20
Europe
TRO19622,
TROPHOS
Spinal Muscular Atrophy (SMA)
 
 
ChiCTR1800018378: A clinical trial and application of pulsed radiofrequency treatment of genicular nerves for muscle atrophy in knee osteoarthritis

Not yet recruiting
1
60
 
pulsed radiofrequency treatment ;Knee pain points lidocaine and diprospan injection
Huadong Hospital Affiliated To Fudan University; Level of the institution：, raise independently
Knee Osteoarthritis
 
 
ChiCTR-IIR-17013983: Effect of Xiaoyu Xiezhuo Yin on Muscular Atrophy Induced by Chronic Renal Failure through IGF-1 / PI3K / Akt Signaling Pathway

Not yet recruiting
1
100
 
Based on Western medicine treatment, add Xiaoyu Xiezhuo drink. ;Only Western medicine treatment
Zhejiang Provincial Hospital of TCM; Zhejiang Provincial Hospital of TCM, Zhejiang Provincial Hospital of TCM
chronic kidney disease
 
 
EXG001-307-101, NCT06576388: Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

Completed
1
2
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Type I Spinal Muscular Atrophy
09/23
01/24
NCT04708847: A Clinical Pharmacology Study to Evaluate the Effect of GYM329 on Disuse Muscle Atrophy in Healthy Volunteer

Completed
1
30
Europe
GYM329, Placebo
Chugai Pharmaceutical
Healthy Volunteers
06/22
02/23
NCT04718181: Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

Completed
1
131
US
risdiplam, Evrysdi, omeprazole, Non-investigational medicinal product
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/23
01/23
ACTRN12622000964718: A Phase 1 trial assessing the Safety, Tolerability, and Pharmacokinetics of NIDO-361 in Healthy Male Subjects

Completed
1
64
 
Nido Biosciences, Inc., Nido Biosciences, Inc.
Spinal and bulbar muscular atrophy (SBMA)
 
 
NCT05730842: Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers

Completed
1
15
US
EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
Edgewise Therapeutics, Inc.
Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn
04/23
04/23
ChiCTR2200064801: The effect mechanism of exercise through the intestinal - muscle axis to intervene the aging muscle atrophy

Not yet recruiting
1
400
 
exercise training ;none
Beijing Sport University; Beijing Sport University, Special funds from the central government
sarcopenia
 
 
ChiCTR2200063230: Single arm, single center, high-dose clinical study on the safety, tolerance and efficacy of GC101 adeno-associated virus injection in patients with type 1 spinal muscular atrophy

Recruiting
1
4
 
GC101 intrathecal injection
The Seventh Medical Center of PLA General Hospital ; The Seventh Medical Center of PLA General Hospital, fund projects
type 1 spinal muscular atrophy
 
 
NCT04057833: E-CEL UVEC Cells as an Adjunct Cell Therapy for the Arthroscopic Rotator Cuff Repair in Adults

Active, not recruiting
1
20
US
E-CEL UVEC
Hospital for Special Surgery, New York, Angiocrine Bioscience
Rotator Cuff Tears, Muscle Atrophy or Weakness, Tendon Rupture - Shoulder, Safety Issues
10/24
10/24
ChiCTR2200063672: EC50 of propofol with target controlled infusion for intrathecal injection of Nusinersen in children with spinal muscular atrophy

Not yet recruiting
1
30
 
Target controlled infusion of propofol for intravenous anesthesia
The First Affiliated Hospital of Fujian Medical University; The First Affiliated Hospital of Fujian Medical University, self-raised
Spinal muscular atrophy
 
 
ChiCTR2100054441: Single arm and single center clinical study on safety, tolerance and efficacy of GC101 injection in patients with type 2 and 3 spinal muscular atrophy

Completed
1
10
 
GC101 intrathecal injection
Seventh Medical Center of PLA General Hospital; Seventh Medical Center of PLA General Hospital, Fund projects
type 2 and 3 spinal muscular atrophy
 
 
NCT03107884: Role of Metformin on Muscle Health of Older Adults

Active, not recruiting
1
64
US
Metformin (Bed Rest), Metformin hydrochloride, Fortamet, glucophage, Glumetza, Riomet, Placebo (Bed Rest), Metformin (2 week run-in only), Glucophage, Placebo (2 week run-in only)
University of Utah
Muscle Atrophy, Insulin Resistance
11/25
11/25
NCT06185179: Metformin and Muscle Recovery

Recruiting
1
50
US
Metformin, Glucophage, Fortamet, Glumetza, Riomet, Placebo
University of Utah
Muscle Atrophy or Weakness
05/29
05/30
PIERRE-PK, NCT06555419: A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy

Recruiting
1
55
US
Nusinersen, BIIB058, ThecaFlex DRx System
Biogen
Muscular Atrophy, Spinal
12/25
12/25
NCT06888661: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy

Enrolling by invitation
1
4
RoW
EXG001-307 injection
Hangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy (SMA)
03/26
12/26
NCT05575011 / 2022-000956-12: A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

Recruiting
1
62
Europe, Canada, RoW
BIIB115, BIIB115-Matching Placebo
Biogen, Biogen Idec Research Limited
Healthy Volunteer, Muscular Atrophy, Spinal
09/27
09/27
ChiCTR2500095667: An open label, single arm IIT clinical study evaluating the safety, tolerability, and preliminary efficacy of GCB-001 in the treatment of patients with delayed onset type 2 SMA who can sit alone but cannot walk

Not yet recruiting
1
6
 
Intrathecal injection Low Dosing GCB-001; Intrathecal injection High Dosing GCB-001
The Children's Hospital of Zhejiang University School of Medicine; The Children's Hospital of Zhejiang University School of Medicine, Enterprise funding
Spinal muscular atrophy
 
 
ACTRN12623000265673: Foot muscle measures of strength and cross-sectional area by ultrasound in the diabetic population.

Not yet recruiting
N/A
21
 
The University of Sydney , N/A
Diabetic peripheral neuropathy , Diabetic foot changes , Intrinsic foot muscle atrophy , Intrinsic foot muscle weakness
 
 
NCT06877689: EAP of Apitegromab for Patients With Spinal Muscular Atrophy

Available
N/A
NA
Apitegromab, SRK-015
Scholar Rock, Inc.
SMA
 
 
NCT00443066: Clinical Study of Spinal Muscular Atrophy

Completed
N/A
120
US
Columbia University, The Spinal Muscular Atrophy Foundation
Spinal Muscular Atrophy
08/11
08/11
ChiCTR-IPR-15007273: The effect of nutrition support combining with early rehabilitation on muscle mass and strength in ICU patients

Recruiting
N/A
60
 
early rehabilitation ;routine treatment
Beijing Tongren Hospital, Capital medical university; Beijing Tongren Hospital, Capital medical university, Self-raised funds
critical illness muscle atrophy
 
 
NCT06666816: Observational Study to Observe Variations of Gait Parameters in Patients with Neuromuscular Diseases

Recruiting
N/A
120
Europe
inapplicable
IRCCS Eugenio Medea
Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
12/26
12/26
SMA, NCT03217578: Neonatal Spinal Muscular Atrophy () Screening

Recruiting
N/A
250000
RoW
SMA Newborn Screening
Kaohsiung Medical University Chung-Ho Memorial Hospital, The Chinese Foundation of Health, Taipei Institute of Pathology
Spinal Muscular Atrophy
12/30
12/30
ChiCTR1800019722: Application of MUNIX in evaluating the surgical effect of patients with cervical spondylosis muscular atrophy

Recruiting
N/A
70
 
operation ;operation ;non-operation
Huashan Hospital, Fudan University; Huashan Hospital, Fudan University, Shanghai City Health System of the Second Batch of Important Diseases Combined Project (2014ZYJ0008)
Cervical Spondylotic Amyotrophy
 
 
ChiCTR1900026896: Effect of acupuncture regulating macrophage polarization to alleviate sepsis-induced muscle atrophy: a prospective study

Suspended
N/A
98
 
acupuncture+basic treatment ;basic treatment
Department of Intensive Care Unit, the First Affiliated Hospital of Guangzhou University of Chinese Medicine; The First Affiliated Hospital of Guangzhou University of Chinese Medicine, Fund of Guangdong Provincial Traditional Chinese Medicine Bureau Research Project, China (20192018), Foundation of Innovative Development Project (2017QN05)
Sepsis-induced myopathy
 
 
 

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