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Clinical trials for Congenital muscular dystrophy

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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 5 of 5.
    « Previous 1  2  3  4  5 
    EudraCT Number: 2020-004803-15 Sponsor Protocol Number: 5051-102 Start Date*: 2023-09-18
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label Extension Study for Patients with Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
    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: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 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: 2013-004427-37 Sponsor Protocol Number: ESPP001 Start Date*: 2014-10-01
    Sponsor Name:PARENT PROJECT ONLUS
    Full Title: Multicentre, randomised, double-blind, dose titration design in patients with Duchenne muscular dystrophy to evaluate the efficacy and the tolerability of the combinations of Ibuprofen (200 mg bid)...
    Medical condition: Restrictive respiratory syndrome with frequent pulmonary infections and cardiomyopathy. wasting of skeletal muscle, severe local inflammation and, at least initially, muscle regeneration.
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10064571 Gene mutation PT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    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: 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: 2020-002093-27 Sponsor Protocol Number: GNT-016-MDYF Start Date*: 2020-11-30
    Sponsor Name:Genethon
    Full Title: Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple b...
    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: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-004740-36 Sponsor Protocol Number: PTCEMF-GD-004 Start Date*: 2019-10-14
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I
    Medical condition: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10028356 Muscular dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) FR (Completed) DK (Completed)
    Trial results: View results
    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: 2017-002213-60 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
    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 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001181-15 Sponsor Protocol Number: FIS-002-2019 Start Date*: 2019-09-17
    Sponsor Name:Fulcrum Therapeutics, Inc.
    Full Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD)
    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) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-000601-36 Sponsor Protocol Number: GUP15004 Start Date*: 2016-07-26
    Sponsor Name:FONDAZIONE SERENA ONLUS
    Full Title: CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1
    Medical condition: Myotonic Dystrophy type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000305-79 Sponsor Protocol Number: A083-04 Start Date*: 2019-05-14
    Sponsor Name:Acceleron Pharma Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with C...
    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: 2019-004426-24 Sponsor Protocol Number: TREAT-LMNA Start Date*: 2020-07-06
    Sponsor Name:AZIENDA OSPEDALIERO-UNIVERSITARIA PISANA
    Full Title: Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers.
    Medical condition: Congenital Muscolar distrophy LMNA related
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004859 10003718 Atrophy skeletal muscle LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004602-94 Sponsor Protocol Number: BIO101_CL04 Start Date*: 2020-03-27
    Sponsor Name:Biophytis
    Full Title: A 3-part, Randomized, Double Blind, Adaptive Seamless Phase 1-3 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO101 in Non-Ambulatory Patients with a Geneticall...
    Medical condition: Duchenne Muscular Dystrophy regardless the genotype
    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: Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-002688-32 Sponsor Protocol Number: 1602T0832 Start Date*: 2017-11-07
    Sponsor Name:Shionogi Ltd.
    Full Title: A Phase 3, Multicenter, Randomized, Double-blind Study of a Single Dose of S-033188 Compared with Placebo or Oseltamivir 75 mg Twice Daily for 5 Days in Patients with Influenza at High Risk of Infl...
    Medical condition: Influenza
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004862 10022005 Influenza viral infections HLT
    20.1 100000004862 10022002 Influenza A virus infection LLT
    20.1 100000004862 10022003 Influenza B virus infection LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) LV (Completed) HU (Completed) GB (Completed) PL (Completed) BG (Completed) ES (Completed) BE (Completed) RO (Completed)
    Trial results: View results
    EudraCT Number: 2012-002933-12 Sponsor Protocol Number: 11/0090 Start Date*: 2014-04-07
    Sponsor Name:University College London,
    Full Title: A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdom...
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
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    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
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