Online Trial Tracker - Larvol Sigma

Trial + Data / Events

Status

Region

Interventions

Sponsor

Conditions

Primary compl

Study compl

Muscular Atrophy

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

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

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

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]

2021-003474-31: Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)

Not yet recruiting

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

Europe, Canada, US, RoW

OAV101

Novartis Pharmaceuticals

Spinal Muscular Atrophy

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

292

Europe, Canada, Japan, US, RoW

nusinersen, ISIS 396443, Spinraza, BIIB058, IONIS SMN Rx, ISIS SMNRx

Biogen

Spinal Muscular Atrophy

08/23

DEVOTE, NCT04089566 / 2019-002663-10: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Completed

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

Active, not recruiting

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

10/24

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

Active, not recruiting

Europe, Canada, Japan, US, RoW

OAV101, AVXS-101, Zolgensma

Novartis Pharmaceuticals

Spinal Muscular Atrophy

12/24

HOPE-3, NCT05126758: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Recruiting

102

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

12/24

12/26

STEER, NCT05089656: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Active, not recruiting

125

Europe, US, RoW

OAV101, Zolgensma, AVXS-101, Sham control

Novartis Pharmaceuticals

Type 2 Spinal Muscular Atrophy

12/24

02/25

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

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

ONWARD, NCT04729907 / 2020-004708-32: Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen

Enrolling by invitation

145

Europe, Canada, Japan, US, RoW

Nusinersen, BIIB058, Spinraza

Biogen, Biogen Idec Research Limited

Muscular Atrophy, Spinal

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

260

Europe, US

Apitegromab

Scholar Rock, Inc., Scholar Rock, Inc.

Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin

11/26

01/27

ASCEND, NCT05067790 / 2021-001294-23: A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam

Recruiting

Europe, Japan, US

Nusinersen, BIIB058, Spinraza

Biogen

Spinal Muscular Atrophy

06/27

NCT04042025 / 2019-002611-26: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active, not recruiting

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

NCT05335876: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Recruiting

260

Europe, Canada, Japan, US, RoW

onasemnogene abeparvovec, Zolgensma

Novartis Pharmaceuticals

Spinal Muscular Atrophy (SMA)

10/39

NCT04456530: Use of Testosterone to Prevent Post-Surgical Muscle Loss - Pilot Study

Enrolling by invitation

2/3

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

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

Recruiting

2/3

259

Europe, Canada, Japan, US, RoW

RO7204239, Placebo, Risdiplam, RO7034067, Evrysdi

Hoffmann-La Roche

Spinal Muscular Atrophy (SMA)

06/26



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