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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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    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    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 3 of 6.
    « Previous 1  2  3  4  5  6  Next»
    EudraCT Number: 2018-003618-41 Sponsor Protocol Number: MOM-M281-005 Start Date*: 2019-06-05
    Sponsor Name:Momenta Pharmaceuticals, Inc.
    Full Title: An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients with Generalized Myasthenia Gravis
    Medical condition: Generalized Myasthenia gravis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10028417 Myasthenia gravis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Temporarily Halted) DE (Prematurely Ended) BE (Completed) ES (Prematurely Ended) PL (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-001637-88 Sponsor Protocol Number: 2014-650 Start Date*: 2014-08-12
    Sponsor Name:John Vissing
    Full Title: A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disorder type V)
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2022-003126-42 Sponsor Protocol Number: DT-DEC01-DMD-CT-Phase1/2 Start Date*: 2023-12-12
    Sponsor Name:Dystrogen Therapeutics Technology Polska Spółka z Ograniczoną Odpowiedzialnością
    Full Title: A Phase 1/2, Open-Label, Exploratory Clinical Trial to Evaluate the Safety and Efficacy of DT-DEC01 Therapy in Patients with Duchenne Muscular Dystrophy
    Medical condition: Duchenne muscular dystrophy (DMD) is the most common inherited neuromuscular disease that mostly affects boys. DMD is a type of X-linked disease caused by a mutation of the gene at the Xp21 locus t...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-003028-19 Sponsor Protocol Number: PS_KSS_001_2019 Start Date*: 2019-10-30
    Sponsor Name:IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
    Full Title: Clinical-instrumental definition of the phenotypic spectrum, response to treatment and natural history in Pearson and Kearns-Sayre syndrome
    Medical condition: Pearson and Kearns-Sayre syndrome.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10058799 Mitochondrial encephalomyopathy PT
    20.1 10010331 - Congenital, familial and genetic disorders 10058799 Mitochondrial encephalomyopathy PT
    20.1 10010331 - Congenital, familial and genetic disorders 10051403 Mitochondrial DNA deletion PT
    20.1 10010331 - Congenital, familial and genetic disorders 10051403 Mitochondrial DNA deletion PT
    20.0 10010331 - Congenital, familial and genetic disorders 10074070 Mitochondrial encephalopathy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-001762-42 Sponsor Protocol Number: 4658-402 Start Date*: 2020-04-03
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular ...
    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 (Completed) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) ES (Ongoing) SE (Prematurely Ended) DE (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) NO (Trial now transitioned) HU (Trial now transitioned) CZ (Trial now transitioned) SI (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-002247-28 Sponsor Protocol Number: MOM-M281-004 Start Date*: 2019-02-05
    Sponsor Name:Momenta Pharmaceuticals, Inc.
    Full Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults with ...
    Medical condition: Treatment of MG, with an initial focus on patients with gMG treated with M281 and evaluation of the expected reduction of circulating levels of antibodies by blocking IgG recycling, including the p...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10028417 Myasthenia gravis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) PL (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-000778-40 Sponsor Protocol Number: BP39056 Start Date*: 2017-01-05
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY
    Medical condition: Type 1 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
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Prematurely Ended) IT (Completed) BE (Completed) FR (Completed) PL (Completed) HR (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: 2020-003492-18 Sponsor Protocol Number: 232SM404 Start Date*: 2021-06-07
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
    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, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-005749-30 Sponsor Protocol Number: 2015-00887 Start Date*: 2016-06-23
    Sponsor Name:Karolinska Institutet
    Full Title: A randomized, doubleblind, placebo-controlled multicenter trial to evaluate the safety and efficacy of rituximab (Mabthera) in subjects with new onset myasthenia gravis; the RINOMAX study
    Medical condition: New onset myasthenia gravis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: View results
    EudraCT Number: 2014-002627-10 Sponsor Protocol Number: RILUMEX Start Date*: 2015-11-03
    Sponsor Name:FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
    Full Title: A randomized, double-blind, controlled, monocenter, pivotal phase IIb study to evaluate the efficacy and safety of riluzole versus mexiletine in patients with non dystrophic myotonia mutated in SCN...
    Medical condition: NDM patients with mutations in SCN4A or CLCN1 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-000097-35 Sponsor Protocol Number: CCFZ533X2204 Start Date*: 2015-09-02
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A multi-center, randomized, double-blind, placebo-controlled, parallel group study to preliminarily evaluate the safety, tolerability, pharmacokinetics and efficacy of CFZ533 in patients with moder...
    Medical condition: Acquired myasthenia gravis (MG) is an autoimmune disease that leads to fluctuating muscle weakness and fatigue. In the most common cases, muscle weakness is caused by circulating antibodies that bi...
    Disease: Version SOC Term Classification Code Term Level
    18.1 10029205 - Nervous system disorders 10028417 Myasthenia gravis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2016-001384-37 Sponsor Protocol Number: ECU-MG-303 Start Date*: 2020-10-07
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis (gMG)
    Medical condition: Refractory Generalized Myasthenia Gravis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10028417 Myasthenia gravis PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) NL (Ongoing)
    Trial results: View results
    EudraCT Number: 2013-004195-36 Sponsor Protocol Number: 120542 Start Date*: 2014-07-21
    Sponsor Name:University College of London
    Full Title: A randomised, double-blind, placebo-controlled, phase II clinical trial with a cross-over design assessing efficacy of a single dose of bumetamide in reducing focal attack severity in hypokalaemic ...
    Medical condition: Hypokalaemic Periodic Paralysis
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10016208 Familial periodic paralysis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-023161-21 Sponsor Protocol Number: 2010.607/11 Start Date*: 2011-11-23
    Sponsor Name:Hospices Civils de Lyon
    Full Title: Evaluation de la toxine botulinique dans le traitement du trouble salivaire chez le patient atteint de sclérose latérale amyotrophique
    Medical condition: Sclérose Latérale Amyotrophique (SLA)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2024-000422-17 Sponsor Protocol Number: C3671016 Start Date*: 2024-08-26
    Sponsor Name:Pfizer Inc.
    Full Title: A PHASE 1, OPEN-LABEL, AGE DESCENDING, DOSE-FINDING STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND IMMUNOGENICITY OF RESPIRATORY SYNCYTIAL VIRUS PREFUSION F SUBUNIT VACCINE (RSVpreF) IN CHILDREN ...
    Medical condition: Prevention of RSV-associated lower respiratory tract illness in children 2-18 years of age by active immunization
    Disease: Version SOC Term Classification Code Term Level
    21.1 10042613 - Surgical and medical procedures 10066742 Respiratory syncytial virus infection prophylaxis LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-002799-26 Sponsor Protocol Number: Start Date*: 2015-10-27
    Sponsor Name:Papworth Hospital NHS Foundation Trust
    Full Title: TRANSFORM-UK: A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension
    Medical condition: Pulmonary arterial hypertension
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-000160-28 Sponsor Protocol Number: SMA-002 Start Date*: 2018-11-06
    Sponsor Name:CATALYST PHARMACEUTICALS INC.
    Full Title: Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3
    Medical condition: Spinal Muscular Atrophy (SMA) Type 3
    Disease: Version SOC Term Classification Code Term Level
    20.1 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)
    Trial results: (No results available)
    EudraCT Number: 2019-001542-17 Sponsor Protocol Number: T109/2019Xe-SAH Start Date*: 2020-09-17
    Sponsor Name:Timo Laitio
    Full Title: Effect of xenon on brain injury, neurological outcome and survival in patients after aneurysmal subarachnoid hemorrhage
    Medical condition: Acute rupture of cerebral aneurysm, subarachnoid hemorrhage (SAH), unconsciousness, brain injury.
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004852 10008078 Cerebral arterial aneurysm LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-003473-94 Sponsor Protocol Number: UCL-2016-121 Start Date*: 2019-05-20
    Sponsor Name:Cliniques Universitaires Saint-Luc
    Full Title: Tau and Amyloid PET imaging in normal aging, early Alzheimer's disease and related syndroms.
    Medical condition: Alzheimer’s disease (AD)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: BE (Trial now transitioned)
    Trial results: (No results available)
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