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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 2 of 5.
    « Previous 1  2  3  4  5  Next»
    EudraCT Number: 2011-004667-76 Sponsor Protocol Number: Cardoz-004 Start Date*: 2011-12-19
    Sponsor Name:Cardoz AB
    Full Title: An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or...
    Medical condition: Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    14.1 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    14.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: SE (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: 2020-000699-39 Sponsor Protocol Number: FGCL-3019-094 Start Date*: 2021-11-04
    Sponsor Name:FibroGen, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD)
    Medical condition: Ambulatory 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 (Completed) BE (Completed) AT (Prematurely Ended) NL (Completed) IT (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-001194-25 Sponsor Protocol Number: H6D-MC-LVJJ Start Date*: 2013-09-25
    Sponsor Name:Eli Lilly and Company
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    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 Gender: Male
    Trial protocol: GB (Prematurely Ended) IT (Prematurely Ended) DE (Completed) ES (Prematurely Ended) BE (Prematurely Ended) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-005040-10 Sponsor Protocol Number: PRO045-CLIN-01 Start Date*: 2012-12-03
    Sponsor Name:BioMarin Nederland B.V.
    Full Title: A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects with Duchenne muscular dystrophy
    Medical condition: Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping
    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) GB (Completed) IT (Prematurely Ended) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-000951-29 Sponsor Protocol Number: 4658-102 Start Date*: 2017-06-22
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    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: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Completed) DE (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-001910-88 Sponsor Protocol Number: ATYR1940-C-004 Start Date*: 2015-12-17
    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 Limb Girdle and Facioscapulohumeral Musc...
    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: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2017-004625-32 Sponsor Protocol Number: 4045-302 Start Date*: 2019-02-05
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 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, Adults Gender: Male
    Trial protocol: FR (Completed) DE (Completed) SE (Completed) BE (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA) CZ (Completed) PL (Completed) BG (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2017-002686-21 Sponsor Protocol Number: WVE-DMDX51-001 Start Date*: 2018-04-11
    Sponsor Name:Wave Life Sciences Ltd.
    Full Title: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to 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 (Completed) FR (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    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: 2020-000698-26 Sponsor Protocol Number: FGCL-3019-093 Start Date*: 2023-01-17
    Sponsor Name:FibroGen, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)
    Medical condition: Non-ambulatory 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, Adults, Elderly Gender: Male
    Trial protocol: FR (Prematurely Ended) AT (Prematurely Ended) CZ (Prematurely Ended) NL (Prematurely Ended) BE (Prematurely Ended) GB (GB - no longer in EU/EEA) IT (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-002530-50 Sponsor Protocol Number: EspeRare_RIM_001 Start Date*: 2015-11-04
    Sponsor Name:EspeRare
    Full Title: A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy.
    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: ES (Completed) GB (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-023744-33 Sponsor Protocol Number: 2 Start Date*: 2012-11-12
    Sponsor Name:University of Rochester
    Full Title: Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen
    Medical condition: Duchenne muscular dystrophy (DMD)
    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 (GB - no longer in EU/EEA) IT (Ongoing)
    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
    EudraCT Number: 2020-003653-30 Sponsor Protocol Number: NS-065/NCNP-01-211 Start Date*: 2021-06-08
    Sponsor Name:NS Pharma, Inc
    Full Title: A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Con...
    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: IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2011-005042-35 Sponsor Protocol Number: PRO053-CLIN-01 Start Date*: 2013-08-23
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053)...
    Medical condition: Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping
    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: GB (Prematurely Ended) BE (Completed) IT (Prematurely Ended) NL (Prematurely Ended) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2015-004333-27 Sponsor Protocol Number: SMTC11005 Start Date*: 2016-01-19
    Sponsor Name:Summit (Oxford) Limited
    Full Title: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)
    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: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-004901-29 Sponsor Protocol Number: NBK154/1/2020 Start Date*: 2021-06-01
    Sponsor Name:Medical University of Gdańsk
    Full Title: The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, ...
    Medical condition: Duchenne muscular dystrophy Cardiomyopathy Tachycardia
    Disease: Version SOC Term Classification Code Term Level
    27.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-003681-87 Sponsor Protocol Number: BMN-044-201 Start Date*: 2016-01-12
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy
    Medical condition: Duchenne muscular dystrophy
    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, Adults Gender: Male
    Trial protocol: BE (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003195-68 Sponsor Protocol Number: 2015-003195-68 Start Date*: 2015-11-11
    Sponsor Name:The Newcastle upon Tyne NHS Hospitals Foundation Trust
    Full Title: Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy.
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
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