UCSD Altman Clinical and Translational Research Institute
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 0 Trials 
9 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Patel, Ankita
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
Stechschulte, Mariah
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
NCT03981575: Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Recruiting
N/A
700
Europe, Canada, US, RoW
Virginia Commonwealth University, University of Rochester, Stanford University, Ohio State University, University of Florida, University of Iowa, Ludwig-Maximilians - University of Munich, Fondazione Serena Onlus - Centro Clinico NeMO Milano, The Methodist Hospital Research Institute, Radboud University Medical Center, University College London Hospitals, University of California, Los Angeles
Myotonic Dystrophy 1, DM1
10/26
12/26
PIERRE, NCT05866419: Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

Recruiting
N/A
90
Europe, US
ThecaFlex DRx System
Alcyone Therapeutics, Inc, Biogen
Spinal Muscular Atrophy, Spine Deformity, Scoliosis
05/26
03/27
Laverty, Chamindra
EMERGENE, NCT06246513: A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

Recruiting
3
15
Europe, US
SRP-9003, scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec, Glucocorticoid, Prednisone or equivalent
Sarepta Therapeutics, Inc.
Limb-girdle Muscular Dystrophy
01/25
11/29
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
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
NCT03981575: Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Recruiting
N/A
700
Europe, Canada, US, RoW
Virginia Commonwealth University, University of Rochester, Stanford University, Ohio State University, University of Florida, University of Iowa, Ludwig-Maximilians - University of Munich, Fondazione Serena Onlus - Centro Clinico NeMO Milano, The Methodist Hospital Research Institute, Radboud University Medical Center, University College London Hospitals, University of California, Los Angeles
Myotonic Dystrophy 1, DM1
10/26
12/26

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