University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)
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 0 Trials 
10 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Baranello, Giovanni
GUARDIAN, NCT06713135: A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

Recruiting
4
80
Europe, RoW
vamorolone 40 mg/mL oral suspension, vamorolone
Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
09/28
09/28
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
3
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
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
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
NCT05938023: A Study of ATL1102 or Placebo in Participants with Non-ambulatory Duchenne Muscular Dystrophy

Terminated
2
48
Europe, RoW
ATL1102 25mg, ATL1102 50mg, Placebo
Percheron Therapeutics
Duchenne Muscular Dystrophy
11/24
01/25
XLMTM, NCT04915846: Tamoxifen Therapy for Myotubular Myopathy

Terminated
1/2
6
Europe, Canada, US
ApoTamox 10mg, Tamoxifen Citrate, Placebo
James Dowling, Canadian Institutes of Health Research (CIHR), Cures Within Reach, The Joshua Frase Foundation USA, Will Cure USA, Mogford Campbell Family Chair Fund, Myotubular Trust, Great Ormond Street Hospital Charity, Sparks
X Linked Myotubular Myopathy
05/24
05/24
NCT06280209: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants with Duchenne Muscular Dystrophy

Recruiting
1/2
18
Europe, RoW
BMN 351
BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
12/25
12/25
NatHis-NM-MDUK, NCT06670378: Natural History Study for Patients With Nemaline Myopathy in the UK

Recruiting
N/A
45
Europe
University of Oxford, Muscular Dystrophy UK
Nemaline Myopathy
08/29
08/29
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
ExSpAnD SMA, NCT05954455: Exploring Bulbar Function, Speech And Communication Development in SMA Type 1

Recruiting
N/A
30
Europe
Institute of Child Health, Great Ormond Street Hospital for Children NHS Foundation Trust
SMA1
09/26
12/26

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