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Clinical trials for Myopathy

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
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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   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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    72 result(s) found for: Myopathy. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2022-003359-33 Sponsor Protocol Number: MABS06 Start Date*: 2023-06-21
    Sponsor Name:MUMC
    Full Title: Assess efficacy of intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers
    Medical condition: Mitochondrial myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002855-40 Sponsor Protocol Number: REN001-201 Start Date*: 2021-04-15
    Sponsor Name:Reneo Pharma Ltd.
    Full Title: A DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY TO EVALUATE THE EFFICACY AND SAFETY OF 24 WEEKS TREATMENT WITH REN001 IN PATIENTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)
    Medical condition: Primary Mitochondrial Myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) CZ (Completed) DK (Completed) HU (Completed) BE (Completed) IT (Completed) ES (Ongoing) NL (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2014-003501-15 Sponsor Protocol Number: 408-C-1403 Start Date*: 2015-03-05
    Sponsor Name:Reata Pharmaceuticals, Inc.
    Full Title: A PHASE 2 STUDY OF THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF RTA 408 IN THE TREATMENT OF MITOCHONDRIAL MYOPATHY
    Medical condition: Mitochondrial myopathy as evidenced by the following 2 criteria (must meet both): a. Have a history of exercise intolerance with or without weakness and/or progressive exercise intolerance (in whi...
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2016-000360-42 Sponsor Protocol Number: UX001-CL302 Start Date*: 2016-08-02
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myo...
    Medical condition: GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000167584 10075048 Hereditary inclusion body myopathy LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10077945 GNE myopathy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) BG (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-003471-34 Sponsor Protocol Number: REN001-202 Start Date*: 2022-04-19
    Sponsor Name:Reneo Pharma Ltd.
    Full Title: AN OPEN-LABEL, MULTI-CENTRE STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF REN001 IN SUBJECTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)
    Medical condition: Primary Mitochondrial Myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) ES (Prematurely Ended) DK (Prematurely Ended) BE (Completed) HU (Prematurely Ended) NL (Ongoing) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-002447-15 Sponsor Protocol Number: SPIMM-301 Start Date*: 2018-06-22
    Sponsor Name:Stealth BioTherapeutics Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondr...
    Medical condition: Primary Mitochondrial Myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) DK (Prematurely Ended) DE (Completed) HU (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-001277-23 Sponsor Protocol Number: ARGX-113-2007 Start Date*: 2022-10-26
    Sponsor Name:argenx BV
    Full Title: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Phar...
    Medical condition: Active Idiopathic Inflammatory Myopathy (IIM)
    Disease: Version SOC Term Classification Code Term Level
    24.1 10028395 - Musculoskeletal and connective tissue disorders 10085970 Idiopathic inflammatory myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned) BE (Trial now transitioned) LT (Trial now transitioned) SE (Trial now transitioned) DE (Trial now transitioned) HU (Trial now transitioned) IE (Trial now transitioned) AT (Trial now transitioned) ES (Trial now transitioned) PT (Trial now transitioned) IT (Trial now transitioned) SK (Trial now transitioned) PL (Trial now transitioned) BG (Trial now transitioned) GR (Trial now transitioned) CZ (Trial now transitioned) NL (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-004553-41 Sponsor Protocol Number: UX001-CL203 Start Date*: 2017-01-05
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 2 Open-label study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (also known as Hereditary Inclusion Body Myopathy (HIBM)) patients wit...
    Medical condition: GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka’s disease, or quadriceps sparing myopathy (QSM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10075048 Hereditary inclusion body myopathy LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10077945 GNE myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-001497-29 Sponsor Protocol Number: RA101495-02.202 Start Date*: 2019-10-17
    Sponsor Name:Ra Pharmaceuticals, Inc.
    Full Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Immune-Mediated Necrotizing Myopathy
    Medical condition: Immune-Mediated Necrotizing Myopathy
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004859 10072156 Immune-mediated necrotizing myopathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-000508-24 Sponsor Protocol Number: 7406 Start Date*: 2015-06-12
    Sponsor Name:Newcastle Upon Tyne Hospitals NHS Trust
    Full Title: A Feasibility Study of Bezafibrate in Mitochondrial Myopathy
    Medical condition: Mitochondrial myopathy due to mt.3243A>G mutation
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-000391-15 Sponsor Protocol Number: AIFA-2016-02364869 Start Date*: 2018-06-05
    Sponsor Name:Azienda Unità Sanitaria Locale - IRCCS di Reggio Emilia
    Full Title: Multicentric, open-label, prospective study of subcutaneous tocilizumab in adult patients with refractory myositis
    Medical condition: Myositis (dermatomyositis [DM] or polymyositis [PM])
    Disease: Version SOC Term Classification Code Term Level
    20.0 10040785 - Skin and subcutaneous tissue disorders 10012503 Dermatomyositis PT
    20.0 10028395 - Musculoskeletal and connective tissue disorders 10028653 Myositis PT
    20.0 10028395 - Musculoskeletal and connective tissue disorders 10036102 Polymyositis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-005432-33 Sponsor Protocol Number: UX001-CL301 Start Date*: 2016-06-07
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion...
    Medical condition: GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka’s disease, or quadriceps sparing myopathy (QSM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075048 Hereditary inclusion body myopathy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BG (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2006-005942-35 Sponsor Protocol Number: 1413 Start Date*: 2007-03-14
    Sponsor Name:POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI
    Full Title: Simvastatin therapy in IBM
    Medical condition: inclusion body myositis
    Disease: Version SOC Term Classification Code Term Level
    6.1 10028641 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-005274-31 Sponsor Protocol Number: 2380/2007 Start Date*: 2008-12-02
    Sponsor Name:AZIENDA OSPEDALIERA PISANA
    Full Title: Efficacy assessment of tetracycline in patients with progressive external ophthalmoplegia.
    Medical condition: Progressive external ophthalmoplegia.
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036802 Progressive external ophthalmoplegia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-002969-19 Sponsor Protocol Number: REN001-101 Start Date*: 2019-01-29
    Sponsor Name:Reneo Pharma Ltd.
    Full Title: An open-label study to evaluate the safety and tolerability of 12 weeks treatment with oral REN001 in patients with primary mitochondrial myopathy (PMM), with an optional extension of treatment
    Medical condition: Primary mitochondrial myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-001192-48 Sponsor Protocol Number: 15-027 Start Date*: 2019-03-04
    Sponsor Name:CHU CAEN
    Full Title: Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes.
    Medical condition: Patients atteints d’une DMOP et présentant un ptosis
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10010498 Congenital hereditary muscular dystrophy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-004608-32 Sponsor Protocol Number: DYN101-C102 Start Date*: Information not available in EudraCT
    Sponsor Name:Dynacure
    Full Title: A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutati...
    Medical condition: Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutations in the myotubularin1 (MTM1) or dynamin 2 (DNM2) gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004859 10028640 Myopathies HLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-001094-25 Sponsor Protocol Number: PHEMI Start Date*: 2019-09-12
    Sponsor Name:FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
    Full Title: Phenylbutyrate Therapy in Mitochondrial Disease with lactic acidosis: an opel label clinical trial in MELAS and PHD deficiency patients
    Medical condition: Melas Syndrome and PDH deficency enchephalopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10053872 MELAS syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10053872 MELAS syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10062950 Leigh syndrome LLT
    Population Age: Children, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-005900-15 Sponsor Protocol Number: P051204 Start Date*: 2007-07-26
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: FORCE : rituximab (anticorps monoclonal anti-CD20 dépléteur des lymphocytes B) pour le traitement des myopathies inflammatoires réfractaires avec auto-anticorps spécifiques et des myasthénies génér...
    Medical condition: Myopathies inflammatoires réfractaires associés à des auto-AC spécifiques Myasthénies généralisées
    Disease: Version SOC Term Classification Code Term Level
    8.1 10028641 Myopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-003907-16 Sponsor Protocol Number: SPIMD-301 Start Date*: 2022-03-02
    Sponsor Name:Stealth BioTherapeutics Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondr...
    Medical condition: Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10010331 - Congenital, familial and genetic disorders 10052635 Cytoplasmic disorders congenital HLGT
    20.0 10010331 - Congenital, familial and genetic disorders 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) NO (Completed) ES (Completed) IT (Completed) NL (Completed) DE (Completed)
    Trial results: (No results available)
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