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Clinical trials for Motor Neuron Disease

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   44346   clinical trials with a EudraCT protocol, of which   7374   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 ).  
     
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    189 result(s) found for: Motor Neuron Disease. Displaying page 8 of 10.
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    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: 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
    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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 2011-005042-35 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: 2007-005478-29 Sponsor Protocol Number: PTC124-GD-007-DMD Start Date*: 2008-03-17
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013801 Duchenne muscular dystrophy LLT
    9.1 10059117 Becker's muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Completed) DE (Completed) SE (Completed) GB (Completed) BE (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-007648-32 Sponsor Protocol Number: PTC124 GD 007e DMD Start Date*: 2009-04-14
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013801 Duchenne muscular dystrophy LLT
    9.1 10059117 Becker's muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed) FR (Completed) DE (Completed) SE (Prematurely Ended) GB (Prematurely Ended) ES (Completed) IT (Prematurely Ended)
    Trial results: View results
    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: 2009-013169-24 Sponsor Protocol Number: PTC124-GD-008-DMD Start Date*: 2010-01-19
    Sponsor Name:PTC Therapeutics Inc
    Full Title: A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy
    Medical condition: Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    12.0 10013801 Duchenne muscular dystrophy LLT
    12.0 10059117 Becker's muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
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