University of Iowa, Stead Family Children's Hospital
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 15 Trials 
22 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
Subramony, SH
STRIDE, NCT04535609 / 2020-002855-40: An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

Completed
2
213
Europe, Canada, US, RoW
Mavodelpar, REN001, Placebo
Reneo Pharma Ltd
Primary Mitochondrial Myopathy
09/23
10/23
NCT03487367: Clinical Trial Readiness for SCA1 and SCA3

Active, not recruiting
N/A
200
US
The Methodist Hospital Research Institute, University of Michigan, University of Minnesota, University of Utah, University of California, Los Angeles, University of Chicago, University of South Florida, Harvard University, Johns Hopkins University, University of Florida, Columbia University, Emory University, University of California, San Francisco, University of Alabama at Birmingham, University of Colorado, Denver, University of Rochester, Stanford University, Northwestern University, German Center for Neurodegenerative Diseases (DZNE), Institut de Recherche sur la Moelle épinière et l'Encéphale, University of Pennsylvania, Ohio State University, University of Iowa
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
12/23
12/23
NCT01060371: Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

Recruiting
N/A
800
US
All Participants
University of Florida, National Ataxia Foundation, University of California, Los Angeles
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
05/24
05/24
NCT06496451: Frataxin mRNA in Biofluids

Recruiting
N/A
10
US
Spinal tap for CSF collection, Venipuncture for blood sample
University of Florida, Friedreich's Ataxia Research Alliance
Friedreich Ataxia
07/25
07/25
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
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
McDaniel, Corey
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
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

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