Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Dystrophin

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   44335   clinical trials with a EudraCT protocol, of which   7366   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
     
    69 result(s) found for: Dystrophin. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2019-001767-67 Sponsor Protocol Number: PTC124-GD-045-DMD Start Date*: 2020-01-30
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment with Ataluren
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001691-11 Sponsor Protocol Number: PTC124-GD-046-DMD Start Date*: 2020-03-06
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001825-28 Sponsor Protocol Number: R119513 Start Date*: 2019-09-24
    Sponsor Name: The University of Manchester
    Full Title: Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscul...
    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: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-000078-27 Sponsor Protocol Number: SRP-9001-102 Start Date*: 2022-11-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
    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
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-003407-15 Sponsor Protocol Number: SRP-9001-104 Start Date*: 2023-08-08
    Sponsor Name:Sarepta Therapeutics, Inc
    Full Title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-exis...
    Medical condition: Duchenne muscular dystrophy
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: ES (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2018-004009-22 Sponsor Protocol Number: WVE-DMDX51-003 Start Date*: Information not available in EudraCT
    Sponsor Name:Wave Life Sciences UK Limited
    Full Title: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory 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: FR (Ongoing) GB (Prematurely Ended) SE (Prematurely Ended) NL (Ongoing) BE (Prematurely Ended) PL (Prematurely Ended) CZ (Prematurely Ended) DE (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-005001-39 Sponsor Protocol Number: 4658-us-202 Start Date*: 2017-03-16
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-000077-83 Sponsor Protocol Number: SRP-9001-101 Start Date*: 2022-11-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Systemic gene delivery Phase I/IIa clinical trial for Duchenne muscular dystrophy using rAAVrh74.MHCK7.micro-dystrophin (microDys-IV-001)
    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
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2006-003833-33 Sponsor Protocol Number: CRO490 Start Date*: 2007-06-13
    Sponsor Name:Imperial College, London
    Full Title: Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013801 Duchenne muscular dystrophy LLT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2006-003075-12 Sponsor Protocol Number: 290478 Start Date*: Information not available in EudraCT
    Sponsor Name:Department Of Paediatric Cardiology
    Full Title: Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction.
    Medical condition: Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patient...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-002520-33 Sponsor Protocol Number: SNT-II-1 Start Date*: 2005-09-06
    Sponsor Name:Santhera Pharmaceuticals LLC
    Full Title: A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac...
    Medical condition: Duchenne Muscular Dystrophy
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2019-002921-31 Sponsor Protocol Number: C3391003 Start Date*: 2020-09-29
    Sponsor Name:Pfizer Inc.
    Full Title: A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF 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, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) IT (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
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    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)
    EudraCT Number: 2017-004139-35 Sponsor Protocol Number: MNK14112096 Start Date*: 2018-06-12
    Sponsor Name:Mallinckrodt ARD Inc.
    Full Title: A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Mus...
    Medical condition: Duchenne Muscular Distrophy
    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: ES (Prematurely Ended) BG (Prematurely Ended) IT (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: 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
    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: IE (Trial now transitioned) BE (Trial now transitioned) ES (Trial now transitioned)
    Trial results: (No results available)
    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: 2019-003374-91 Sponsor Protocol Number: SRP-9001-301 Start Date*: 2022-09-12
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA...
    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, Under 18 Gender: Male
    Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2009-013762-63 Sponsor Protocol Number: PRO044-CLIN-01 Start Date*: 2010-04-16
    Sponsor Name:Prosensa Therapeutics B.V
    Full Title: A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular d...
    Medical condition: Duchenne Muscular Dystrophy
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: NL (Completed) BE (Completed) SE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-004262-26 Sponsor Protocol Number: VBP15-002 Start Date*: 2017-02-13
    Sponsor Name:ReveraGen BioPharma Inc.
    Full Title: A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular ...
    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
    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.
    1  2  3  4  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-Sat May 10 08:57:58 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA