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Clinical trials for Neuromuscular Diseases

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   44343   clinical trials with a EudraCT protocol, of which   7372   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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    103 result(s) found for: Neuromuscular Diseases. Displaying page 5 of 6.
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    EudraCT Number: 2015-001431-20 Sponsor Protocol Number: CHDR1417 Start Date*: 2015-08-17
    Sponsor Name:Centre for Human Drug Research
    Full Title: A randomized, double blind, double-dummy placebo controlled, 3-way cross-over study to determine the test-retest reliability of, and the effect of oral retigabine and riluzole on, peripheral motor ...
    Medical condition: Amytrophic Lateral Sclerosis
    Disease: Version SOC Term Classification Code Term Level
    18.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2014-003644-12 Sponsor Protocol Number: 2014-003644-12 Start Date*: 2015-02-20
    Sponsor Name:Neuromuscular Research Unit, Rigshospitalet
    Full Title: The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V)
    Medical condition: McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10026970 McArdles disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10026969 McArdle's disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10018462 Glycogen storage disease type V PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-004153-17 Sponsor Protocol Number: 20171012 Start Date*: 2018-05-03
    Sponsor Name:Copenhagen Neuromuscular Center
    Full Title: Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest an...
    Medical condition: Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also called: glycogen storage disease Type VII or phosphofructokinase deficiency. ...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053241 Glycogen storage disease type VII PT
    20.1 10010331 - Congenital, familial and genetic disorders 10053250 Glycogen storage disease type III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10053255 Tarui disease LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10016983 Forbes' disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Prematurely Ended)
    Trial results: View results
    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: 2009-017805-13 Sponsor Protocol Number: NGAM-03 Start Date*: 2011-07-14
    Sponsor Name:Octapharma AG
    Full Title: "PROSPECTIVE, PARALLEL GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED, MULTICENTER, ADAPTIVE, TWO-STAGE PHASE II/III STUDY EVALUATING SAFETY AND EFFICACY OF THREE DIFFERENT DOSAGES OF NEWGAM I...
    Medical condition: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10029205 - Nervous system disorders 10057645 Chronic inflammatory demyelinating polyradiculoneuropathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Prematurely Ended) RO (Prematurely Ended) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-002822-21 Sponsor Protocol Number: ARCHIE001 Start Date*: 2013-10-10
    Sponsor Name:University of Oxford
    Full Title: The early use of Antibiotics for at Risk CHildren with InfluEnza in primary care (ARCHIE): a double-blind randomised placebo-controlled trial
    Medical condition: Influenza or influenza-like illness
    Disease: Version SOC Term Classification Code Term Level
    16.0 10021881 - Infections and infestations 10022000 Influenza PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-002269-23 Sponsor Protocol Number: COLELF17 Start Date*: 2018-05-22
    Sponsor Name:AZIENDA OSPEDALIERA UNIVERSITARIA INTEGRATA VERONA
    Full Title: PHARMACOKINETICS OF COLISTIN IN THE ELF AND THE PLASMA OF MDR-GNB OR XDR- GNB VAP OR VAT OR HCAP OR HAP PATIENTS WHO ARE MECHANICALLY VENTILATED: A RANDOMIZED CONTROLLED TRIAL
    Medical condition: ventilator associated pneumonia or ventilator associated tracheobronchitis or health-care associated pneumonia or hospital acquired pneumonia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10021881 - Infections and infestations 10044314 Tracheobronchitis PT
    20.1 10021881 - Infections and infestations 10035701 Pneumonia gram-negative bacterial NOS LLT
    22.0 100000004855 10047263 Ventilation pneumonitis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-000296-21 Sponsor Protocol Number: APHP191110 Start Date*: 2020-04-09
    Sponsor Name:ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (AP-HP)
    Full Title: Rapid rEcognition of COrticosteroid Resistant or sentive Sepsis RECORDS A Multicentre Concealed‐Allocation Multi-arms Blinded Randomized Controlled Trial to Identify the Best Sepsis Population for ...
    Medical condition: Patients admitted to the ICU proven or suspected infection as the main diagnosis.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004862 10040050 Sepsis NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-004708-32 Sponsor Protocol Number: 232SM302 Start Date*: Information not available in EudraCT
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
    Medical condition: Muscular Atrophy, Spinal
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: EE (Completed) ES (Ongoing) HU (Prematurely Ended) DE (Trial now transitioned) PL (Completed) IT (Trial now transitioned) NL (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-002479-20 Sponsor Protocol Number: 80202135MYG2001 Start Date*: 2022-09-29
    Sponsor Name:Janssen-Cilag Invernational N.V.
    Full Title: An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthe...
    Medical condition: Myasthenia gravis (MG)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10028417 Myasthenia gravis PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) Outside EU/EEA PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-002098-12 Sponsor Protocol Number: 232SM201 Start Date*: 2015-05-12
    Sponsor Name:Biogen Idec Research Limited
    Full Title: An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptom...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-016069-27 Sponsor Protocol Number: DORIPED3003 Start Date*: 2010-05-28
    Sponsor Name:Janssen-Cilag International NV, Turnhoutseweg 30, 2340 Beerse, Belgium
    Full Title: A Prospective, Randomized, Double-Blind, Multicenter Study to Establish the Safety and Tolerability of Doripenem Compared With Cefepime in Hospitalized Children With Bacterial Pneumonia
    Medical condition: Bacterial Pneumonia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10021881 - Infections and infestations 10004051 Bacterial pneumonia, unspecified LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: LT (Prematurely Ended) LV (Prematurely Ended) Outside EU/EEA
    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: 2016-000401-36 Sponsor Protocol Number: DSC/14/2357/48 Start Date*: 2017-03-07
    Sponsor Name:ITALFARMACO S.p.A.
    Full Title: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with 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, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Completed) DE (Completed) NL (Completed) IT (Completed) ES (Completed) FR (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
    EudraCT Number: 2019-003757-28 Sponsor Protocol Number: MEX-NM-301 Start Date*: 2020-06-24
    Sponsor Name:Lupin Europe GmbH
    Full Title: An Open-label, non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children with Myotonic Disorders
    Medical condition: Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2); ICD-10 code G71.1
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004852 10028658 Myotonic disorders LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2019-003758-97 Sponsor Protocol Number: MEX-NM-303 Start Date*: 2020-06-24
    Sponsor Name:Lupin Europe GmbH
    Full Title: Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301 and MEX-NM-302 Studies
    Medical condition: Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2); ICD-10 code G71.1
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004852 10028658 Myotonic disorders LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-002087-12 Sponsor Protocol Number: BN40703 Start Date*: 2018-12-07
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10079419 Spinal muscular atrophy pre-symptomatic LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) PL (Trial now transitioned) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2005-003382-16 Sponsor Protocol Number: RMC1 Start Date*: 2005-11-10
    Sponsor Name:Guy's & St Thomas's NHS Foundation Trust [...]
    1. Guy's & St Thomas's NHS Foundation Trust
    2. King's College London
    Full Title: Pilot randomised controlled trial of methotrexate for chronic inflammatory demyelinating polyradiculoneuropathy (RMC trial).
    Medical condition: Chronic inflammatory demyelinating polyradiculoneuropathy
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-002246-41 Sponsor Protocol Number: BP29420 Start Date*: 2014-10-23
    Sponsor Name:F. Hoffmann-La Roche Ltd.
    Full Title: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF T...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) ES (Completed)
    Trial results: View results
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