Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Congenital muscular dystrophy

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44341   clinical trials with a EudraCT protocol, of which   7370   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    97 result(s) found for: Congenital muscular dystrophy. Displaying page 4 of 5.
    « Previous 1  2  3  4  5  Next»
    EudraCT Number: 2016-001615-21 Sponsor Protocol Number: B5161004 Start Date*: 2016-12-12
    Sponsor Name:Pfizer Inc.
    Full Title: A Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy
    Medical condition: Duchenne's Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: GB (Prematurely Ended) IT (Completed) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-000975-34 Sponsor Protocol Number: WVE-DMDX51-002 Start Date*: 2018-10-25
    Sponsor Name:Wave Life Sciences Ltd.
    Full Title: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Prematurely Ended) FR (Ongoing) NL (Completed) BE (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-001654-18 Sponsor Protocol Number: WN40227 Start Date*: 2017-08-12
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: SE (Prematurely Ended) DE (Prematurely Ended) BE (Completed) GB (Completed) ES (Prematurely Ended) NL (Prematurely Ended) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-004276-33 Sponsor Protocol Number: ATA-001-FKRP Start Date*: 2022-02-11
    Sponsor Name:Atamyo Therapeutics
    Full Title: A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle ...
    Medical condition: FKRP-related limb-girdle muscular dystrophy (LGMD R9)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10028356 Muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002372-13 Sponsor Protocol Number: SRP-9001-303 Start Date*: 2023-01-19
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: FR (Trial now transitioned) SE (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000464-29 Sponsor Protocol Number: CAT-1004-301 Start Date*: 2019-01-31
    Sponsor Name:Catabasis Pharmaceuticals, Inc.
    Full Title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: DE (Completed) SE (Completed) IE (Completed)
    Trial results: View results
    EudraCT Number: 2017-001223-49 Sponsor Protocol Number: PTC124-GD-041DMD Start Date*: 2018-01-16
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
    Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BG (Completed) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003568-10 Sponsor Protocol Number: VBP15-LTES Start Date*: 2017-11-23
    Sponsor Name:ReveraGen BioPharma Inc.
    Full Title: A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: SE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-000401-36 Sponsor Protocol Number: DSC/14/2357/48 Start Date*: 2017-03-07
    Sponsor Name:ITALFARMACO S.p.A.
    Full Title: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Completed) DE (Completed) NL (Completed) IT (Completed) ES (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-004279-30 Sponsor Protocol Number: SNT-III-012-E Start Date*: 2018-06-14
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed) GB (Prematurely Ended) FR (Ongoing) SE (Prematurely Ended) NL (Prematurely Ended) DE (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-004554-42 Sponsor Protocol Number: TAMDMD Start Date*: Information not available in EudraCT
    Sponsor Name:University of Basel Children's Hospital, Division of Neuropediatrics
    Full Title: Tamoxifen in Duchenne muscular dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Tamoxifen in Duchenne muscular dystrophy: A 48-week...
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: DE (Prematurely Ended) GB (GB - no longer in EU/EEA) FR (Completed) ES (Prematurely Ended) NL (Prematurely Ended) BE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-002072-92 Sponsor Protocol Number: B5161002 Start Date*: 2014-12-30
    Sponsor Name:Pfizer Inc. 235 East 42nd Street, New York, NY 10017
    Full Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With ...
    Medical condition: Duchenne's Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Prematurely Ended) IT (Prematurely Ended) PL (Prematurely Ended) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-003257-15 Sponsor Protocol Number: A083-02 Start Date*: 2018-04-12
    Sponsor Name:Acceleron Pharma Inc.
    Full Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
    Medical condition: Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-004263-38 Sponsor Protocol Number: VBP-003 Start Date*: 2017-02-13
    Sponsor Name:ReveraGen BioPharma Inc.
    Full Title: A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: SE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2022-003065-38 Sponsor Protocol Number: 1102-DMD-Pre-CT03 Start Date*: 2023-04-13
    Sponsor Name:Antisense Therapeutics Limited
    Full Title: A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by ...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BG (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-001192-48 Sponsor Protocol Number: 15-027 Start Date*: 2019-03-04
    Sponsor Name:CHU CAEN
    Full Title: Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes.
    Medical condition: Patients atteints d’une DMOP et présentant un ptosis
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10010498 Congenital hereditary muscular dystrophy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-001764-11 Sponsor Protocol Number: PTC124-GD-030-DMD Start Date*: 2020-01-21
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
    Medical condition: nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003563-22 Sponsor Protocol Number: CAT-1004-302 Start Date*: 2020-04-23
    Sponsor Name:Catabasis Pharmaceuticals Inc.
    Full Title: An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Prematurely Ended) IE (Prematurely Ended) DE (Prematurely Ended) SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-002704-27 Sponsor Protocol Number: VBP15-004-A4 Start Date*: 2019-07-06
    Sponsor Name:ReveraGen BioPharma, Inc.
    Full Title: A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne...
    Medical condition: Duchenne muscular dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: BE (Completed) SE (Completed) NL (Completed) CZ (Completed) GR (Completed) ES (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2017-001629-41 Sponsor Protocol Number: DSC/15/2357/53 Start Date*: 2018-11-15
    Sponsor Name:ITALFARMACO S.P.A.
    Full Title: A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscu...
    Medical condition: Distrofia Muscolare di Becker (DMB)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Adults Gender: Male
    Trial protocol: NL (Completed) IT (Completed)
    Trial results: View results
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    « Previous 1  2  3  4  5  Next»
    Query did not match any studies.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Wed Jun 11 20:22:25 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA