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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
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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   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).

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    97 result(s) found for: Congenital muscular dystrophy. Displaying page 1 of 5.
    1  2  3  4  5  Next»
    EudraCT Number: 2013-005489-20 Sponsor Protocol Number: PTC124-GD-020e-DMD Start Date*: 2014-08-22
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: SE (Completed) GB (Completed) BE (Completed) DE (Completed) IT (Completed) ES (Completed) CZ (Completed) FR (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2021-000122-10 Sponsor Protocol Number: NS-065/NCNP-01-302 Start Date*: 2021-06-30
    Sponsor Name:NS Pharma, Inc.
    Full Title: A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant 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: NL (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) GR (Trial now transitioned) NO (Trial now transitioned) CZ (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-024566-22 Sponsor Protocol Number: DMD114118 Start Date*: Information not available in EudraCT
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subject...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    12.1 10013801 Duchenne muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Ongoing) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005000-26 Sponsor Protocol Number: 4658-us-201 Start Date*: 2017-03-16
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability, and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administ...
    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: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    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: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-005455-28 Sponsor Protocol Number: WN40226 Start Date*: 2020-09-28
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambula...
    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: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005024-28 Sponsor Protocol Number: 4658-204 Start Date*: 2017-03-28
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
    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, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    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: 2014-003100-78 Sponsor Protocol Number: SMT_C11003 Start Date*: 2014-12-01
    Sponsor Name:Summit (Oxford) Limited
    Full Title: SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patien...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-002115-99 Sponsor Protocol Number: SMTC11002 Start Date*: 2013-08-23
    Sponsor Name:Summit Corporation plc
    Full Title: SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: GB (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
    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: 2010-020069-26 Sponsor Protocol Number: DMD114044 Start Date*: Information not available in EudraCT
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy.
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: DE (Completed) FR (Completed) NL (Completed) BE (Completed) IT (Completed) PL (Completed) CZ (Completed) ES (Completed) NO (Completed) DK (Completed) HU (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005002-19 Sponsor Protocol Number: 4658-301 Start Date*: 2017-03-28
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
    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, Adolescents, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-003605-26 Sponsor Protocol Number: PRO044-CLIN-02 Start Date*: 2014-12-02
    Sponsor Name:Prosensa Therapeutics B.V.
    Full Title: A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IT (Completed) SE (Prematurely Ended) NL (Prematurely Ended) BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-004853-18 Sponsor Protocol Number: PTC124-GD-019-DMD Start Date*: 2012-04-27
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    16.1 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed) SE (Completed) DE (Completed) GB (Completed) ES (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-018412-32 Sponsor Protocol Number: DMD114117 Start Date*: 2010-08-05
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambul...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: DE (Completed) BE (Completed) GB (Completed) NL (Completed) FR (Completed) ES (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-020047-12 Sponsor Protocol Number: P090202 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: Etude de l'effet d'un traitement préventif par Nebivolol sur l'apparition et la progression de la dysfonction cardiaque chez les enfants atteints de dystrophie de Duchenne
    Medical condition: Enfants atteints de dystrophie de Duchenne
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-002210-23 Sponsor Protocol Number: 1.4botox Start Date*: 2014-10-14
    Sponsor Name:Rigshospitalet
    Full Title: Can local intramuscular botulinum toxin improve dysphagia in patients with myopathic dysphagia and constriction of the cricoid muscle?
    Medical condition: Oculopharyngesl muscle dystrophy, inclusion body myositis
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10019897 Hereditary progressive muscular dystrophy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2012-004527-20 Sponsor Protocol Number: PTC124-GD-020-DMD Start Date*: 2013-07-30
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    17.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed) CZ (Completed) SE (Completed) GB (Completed) DE (Completed) IT (Completed) ES (Completed) PL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2019-000337-39 Sponsor Protocol Number: 4658-102-OLE Start Date*: Information not available in EudraCT
    Sponsor Name:Sarepta Therapeutics, Inc
    Full Title: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
    Medical condition: Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    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: FR (Ongoing) BE (Prematurely Ended) GB (GB - no longer in EU/EEA) IT (Prematurely Ended)
    Trial results: View results
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