UCL Institute of Child Health Great Ormond Street
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 5 Trials 
7 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Muntoni, Francesco
MIS51ON, NCT03992430 / 2018-001762-42: A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD)

Active, not recruiting
3
160
Europe, US, RoW
Eteplirsen, AVI-4658, EXONDYS 51, EXONDYS
Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
10/26
10/26
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
NCT03882827: Natural History of Duchenne Muscular Dystrophy

Recruiting
N/A
100
Europe
Genethon
Duchenne Muscular Dystrophy
06/23
06/23
SMA-REACH, NCT03520179: Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)

Recruiting
N/A
600
Europe
Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy
08/23
08/23
NCT04583917: Brain Involvement in Dystrophinopathies Part 1

Completed
N/A
377
Europe
Great Ormond Street Hospital for Children NHS Foundation Trust, Newcastle-upon-Tyne Hospitals NHS Trust, Leiden University Medical Center, Stichting Kempenhaeghe, Region Hovedstadens Apotek, Institut Necker Enfants Malades, Catholic University of the Sacred Heart, Università degli Studi di Ferrara, Universidad Complutense de Madrid
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
06/24
06/24
INC-6603, NCT01203085: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Recruiting
N/A
500
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, Children's Hospital of Philadelphia, University College London Hospitals, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Sydney Children's Hospitals Network, Nemours Children's Clinic
Charcot Marie Tooth Disease
12/24
12/24
INC-6602, NCT01193088: Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Recruiting
N/A
1050
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, University of Pennsylvania, King's College Hospital NHS Trust, Sydney Children's Hospitals Network, Children's Hospital of Philadelphia, University of Miami, Johns Hopkins University, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Cedars-Sinai Medical Center, Nemours Children's Clinic, Stanford University, University of Minnesota, Massachusetts General Hospital, University of Colorado, Denver, Children's National Research Institute, University of Michigan, St. Jude Children's Research Hospital, Connecticut Children's Medical Center, Seattle Children's Hospital, The Hospital for Sick Children
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
12/26
12/26

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