University of Iowa Hospitals and Clinics, Dept of Pediatrics
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 17 Trials 
35 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Mathews, Katherine
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
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
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
REACH CDM, NCT03692312: Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Completed
2/3
56
Europe, Canada, US, RoW
Tideglusib, Placebo
AMO Pharma Limited
Congenital Myotonic Dystrophy
04/23
04/23
REACH CDM X, NCT05004129: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Recruiting
2/3
76
Canada, US, RoW
Tideglusib
AMO Pharma Limited
Congenital Myotonic Dystrophy
03/25
03/25
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
LION-CS101, NCT05230459: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting
1/2
10
US
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Asklepios Biopharmaceutical, Inc.
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
12/28
12/28
MOVE FSHD, NCT04635891: Motor Outcomes to Validate Evaluations in FSHD

Recruiting
N/A
450
Europe, Canada, US
University of Kansas Medical Center, FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche
FSHD
12/26
01/27
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
NCT00313677: Clinical Trial Readiness for the Dystroglycanopathies

Recruiting
N/A
190
US
Katherine Mathews, National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy
07/25
07/26
GRASP-01-003, NCT05618080: LGMD R1 Natural History Study

Recruiting
N/A
100
Europe, US
Virginia Commonwealth University, Nationwide Children's Hospital, Washington University School of Medicine, University of Iowa, University of Florida, University of Minnesota, Newcastle University, University of Kansas Medical Center, University of Colorado, Denver, Indiana CHC (Community Health Clinic), University of California, Irvine
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
05/28
08/28
FA-COMS, NCT03090789: FA Clinical Outcome Measures

Active, not recruiting
N/A
2000
Canada, US, RoW
Friedreich's Ataxia Research Alliance, University of Rochester, Children's Hospital of Philadelphia
Friedreich Ataxia, Neuro-Degenerative Disease
01/30
01/30
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting
N/A
700
Europe, Japan, US, RoW
Prospective observational registry, Zolgensma
Novartis Pharmaceuticals, United BioSource, LLC
Spinal Muscular Atrophy (SMA)
06/38
06/38
UNIFAI, NCT06016946: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Recruiting
N/A
3000
Europe, Canada, US, RoW
Friedreich's Ataxia Research Alliance
Friedreich Ataxia
01/48
01/48
Mercuri, Eugenio
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
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
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
ULYSSES, NCT05933057: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Recruiting
3
138
Europe, Canada
Givinostat, ITF2357, Placebo
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
02/28
02/28
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
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
07/25
07/25
NCT06769633: Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Not yet recruiting
2
18
Europe
Givinostat Hydrochloride, Cohort 1, Givinostat Hydrochloride - Cohort 2, Cohort 2
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
12/29
12/29
CircSMA, NCT05760209: SMN Circular RNAs as Potential New Targets and Biomarkers for SMA

Recruiting
N/A
30
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
07/24
07/24
NCT05779956: Personalized Medicine for SMA: a Translational Project

Recruiting
N/A
90
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Ospedale Pediatrico Bambino Gesu
Spinal Muscular Atrophy
08/24
08/24
NCT05772416: Neonatal Neurological Examination to Detect Infants at Risk

Recruiting
N/A
100
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Neonatal Encephalopathy
08/25
08/25
NCT05768048: Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

Recruiting
N/A
500
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Bambino Gesù Hospital and Research Institute, University of Messina, Gaslini Children's Hospital
Spinal Muscular Atrophy
11/27
11/27
iSMAR, NCT05755451: Natural History of SMA

Recruiting
N/A
1200
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Istituto Giannina Gaslini, Ospedale Pediatrico Bambin Gesù, University of Messina
Muscular Atrophy, Spinal
12/33
12/33
Comi, Giacomo
TRAILBLAZER-ALZ 6, NCT05738486: A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer's Disease

Active, not recruiting
3
800
Europe, US
Donanemab, LY3002813, Placebo
Eli Lilly and Company
Alzheimer's Disease, Dementia, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders
05/24
05/25
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
Fortify, NCT05775848: Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

Active, not recruiting
3
81
Europe, US, RoW
BBP-418 (ribitol), Placebo
ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
07/27
07/27
NCT05973630: ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

Not yet recruiting
1/2
6
Europe, US
ATA-200
Atamyo Therapeutics
LGMD2C
07/26
01/32
NCT04475926: A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

Recruiting
N/A
241
Europe, Canada, US, RoW
Sarepta Therapeutics, Inc.
Limb-girdle Muscular Dystrophy
04/30
04/30
Gibbs, Ciara
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
LION-CS101, NCT05230459: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting
1/2
10
US
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Asklepios Biopharmaceutical, Inc.
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
12/28
12/28
SUNRISE-FA, NCT05445323: Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

Active, not recruiting
1/2
8
US
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Lexeo Therapeutics
Friedreich Ataxia, Cardiomyopathy, Secondary
09/29
09/29
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26

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