- Trials with a EudraCT protocol (6)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
6 result(s) found for: Skeletal muscle fibers.
Displaying page 1 of 1.
| EudraCT Number: 2022-003359-33 | Sponsor Protocol Number: MABS06 | Start Date*: 2023-06-21 | |||||||||||
| Sponsor Name:MUMC | |||||||||||||
| Full Title: Assess efficacy of intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers | |||||||||||||
| Medical condition: Mitochondrial myopathy | |||||||||||||
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| Population Age: Adults | Gender: Male, Female | ||||||||||||
| Trial protocol: NL (Trial now transitioned) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
| EudraCT Number: 2014-001637-88 | Sponsor Protocol Number: 2014-650 | Start Date*: 2014-08-12 | |||||||||||
| Sponsor Name:John Vissing | |||||||||||||
| Full Title: A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) | |||||||||||||
| Medical condition: McArdle disease (Glycogen storage disorder type V) | |||||||||||||
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| Population Age: Adults | Gender: Male, Female | ||||||||||||
| Trial protocol: DK (Completed) | |||||||||||||
| Trial results: View results | |||||||||||||
| EudraCT Number: 2016-005023-92 | Sponsor Protocol Number: 4658-203 | Start Date*: 2017-03-28 | |||||||||||
| Sponsor Name:Sarepta Therapeutics, Inc. | |||||||||||||
| Full Title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | |||||||||||||
| Medical condition: Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping | |||||||||||||
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| Population Age: Children, Under 18 | Gender: Male | ||||||||||||
| Trial protocol: Outside EU/EEA | |||||||||||||
| Trial results: View results | |||||||||||||
| EudraCT Number: 2017-002213-60 | Sponsor Protocol Number: GX1001 | Start Date*: 2019-01-08 | ||||||||||||||||
| Sponsor Name:Solid Biosciences Inc. | ||||||||||||||||||
| Full Title: A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with D... | ||||||||||||||||||
| Medical condition: Duchenne muscular dystrophy | ||||||||||||||||||
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| Population Age: Children, Adolescents, Under 18 | Gender: Male | |||||||||||||||||
| Trial protocol: GB (GB - no longer in EU/EEA) | ||||||||||||||||||
| Trial results: (No results available) | ||||||||||||||||||
| EudraCT Number: 2021-005478-24 | Sponsor Protocol Number: DYNE251DMD201 | Start Date*: Information not available in EudraCT | |||||||||||
| Sponsor Name:Dyne Therapeutics, Inc | |||||||||||||
| Full Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participan... | |||||||||||||
| Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
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| Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
| Trial protocol: IE (Trial now transitioned) BE (Trial now transitioned) ES (Trial now transitioned) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
| EudraCT Number: 2019-000601-77 | Sponsor Protocol Number: 5051-201 | Start Date*: 2019-12-11 | |||||||||||
| Sponsor Name:Sarepta Therapeutics, Inc. | |||||||||||||
| Full Title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | |||||||||||||
| Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
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| Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
| Trial protocol: IE (Prematurely Ended) ES (Temporarily Halted) GB (GB - no longer in EU/EEA) BE (Trial now transitioned) NL (Restarted) DE (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
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