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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:
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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 3 of 5.
    « Previous 1  2  3  4  5  Next»
    EudraCT Number: 2014-005296-81 Sponsor Protocol Number: BMN-051-303 Start Date*: 2016-05-02
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne ...
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-001955-54 Sponsor Protocol Number: BMN-051-302 Start Date*: 2015-12-08
    Sponsor Name:BioMarin Nederland BV
    Full Title: An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: NL (Prematurely Ended) DE (Completed) BE (Completed) ES (Completed)
    Trial results: View results
    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: 2009-012037-30 Sponsor Protocol Number: SNT-III-003 Start Date*: 2009-07-14
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old 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: BE (Completed) DE (Completed) FR (Completed) NL (Completed) SE (Completed) AT (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2025-000201-16 Sponsor Protocol Number: VBP15-006 Start Date*: 2025-05-22
    Sponsor Name:Santhera Pharmaceuticals (Schweiz) AG
    Full Title: A phase II open-label, multiple dose study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of vamorolone in boys ages 2 to <4 years and 7 to <18 yea...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    28.0 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: (No results available)
    EudraCT Number: 2014-003346-27 Sponsor Protocol Number: ATYR1940-C-003 Start Date*: Information not available in EudraCT
    Sponsor Name:ATYR PHARMA, INC.
    Full Title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Fa...
    Medical condition: Early Onset and Other Pediatric Onset 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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2007-004819-54 Sponsor Protocol Number: PRO051-02 Start Date*: 2008-01-22
    Sponsor Name:Prosensa Therapeutics BV
    Full Title: A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscul...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed) NL (Completed) SE (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: 2022-000389-16 Sponsor Protocol Number: 1821-FSH-301 Start Date*: 2022-08-08
    Sponsor Name:Fulcrum Therapeutics
    Full Title: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating 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: FR (Trial now transitioned) NL (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000602-10 Sponsor Protocol Number: SNT-III-012 Start Date*: 2016-11-17
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Ste...
    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: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) SE (Prematurely Ended) GB (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Completed) IT (Prematurely Ended) IE (Completed) HU (Prematurely Ended) BG (Prematurely Ended)
    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: 2011-001266-17 Sponsor Protocol Number: DMD114349 Start Date*: 2011-08-08
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Prematurely Ended) FR (Ongoing) DE (Completed) GB (Prematurely Ended) NL (Prematurely Ended) ES (Prematurely Ended) IT (Prematurely Ended) Outside EU/EEA BG (Prematurely Ended) HU (Prematurely Ended) DK (Prematurely Ended) CZ (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-000624-25 Sponsor Protocol Number: ATYR1940-C-006 Start Date*: 2016-06-22
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy
    Medical condition: Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002076-13 Sponsor Protocol Number: NS-065/NCNP-01-301 Start Date*: 2020-03-02
    Sponsor Name:NS Pharma, Inc.
    Full Title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety 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: GB (GB - no longer in EU/EEA) SE (Completed) ES (Ongoing) NL (Ongoing) GR (Completed) IT (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2007-005808-41 Sponsor Protocol Number: EC 07/90601 Start Date*: 2008-02-25
    Sponsor Name:Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
    Full Title: Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinica...
    Medical condition: Portadores sintomaticos de una mutación en el gen disferlina. Symptomatic carriers of a mutation in the dysferlin gene
    Disease: Version SOC Term Classification Code Term Level
    9.1 10028356 Muscular dystrophy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-001912-36 Sponsor Protocol Number: ATYR1940-C-005 Start Date*: 2015-08-04
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (F...
    Medical condition: Facioscapulohumeral muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-002008-25 Sponsor Protocol Number: 4053-101 Start Date*: 2014-09-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) o...
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 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, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed) IT (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-000397-10 Sponsor Protocol Number: DSC/14/2357/51 Start Date*: 2017-07-31
    Sponsor Name:ITALFARMACO S.p.A.
    Full Title: Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente tr...
    Medical condition: Distrofia Muscolare di Duchenne (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, Elderly Gender: Male
    Trial protocol: IT (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) GB (GB - no longer in EU/EEA) ES (Ongoing) FR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-001753-17 Sponsor Protocol Number: ATYR1940-C-002 Start Date*: 2014-09-19
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Define...
    Medical condition: Facioscapulohumeral muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-001967-38 Sponsor Protocol Number: SMTC11004 Start Date*: 2015-08-03
    Sponsor Name:Summit (Oxford) Limited
    Full Title: A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulati...
    Medical condition: Duchenne muscular dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18, Adults Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
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