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Clinical trials for Congenital myopathy

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   44338   clinical trials with a EudraCT protocol, of which   7368   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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    26 result(s) found for: Congenital myopathy. Displaying page 1 of 2.
    1  2  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: 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: 2020-002855-40 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: 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: 2021-003471-34 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: 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: 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: 2018-002969-19 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: 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)
    EudraCT Number: 2019-001147-51 Sponsor Protocol Number: COMPIS Start Date*: 2020-03-31
    Sponsor Name:Västra götalandsregionen
    Full Title: Congenital myopathy intervention study
    Medical condition: Congenital myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10062547 Congenital myopathy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Trial now transitioned)
    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: 2022-003307-16 Sponsor Protocol Number: OMT28-C0203 Start Date*: 2023-04-18
    Sponsor Name:OMEICOS Therapeutics GmbH
    Full Title: A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD patients with myopathy and/or cardiomyopathy and inflammation (PMD-OPTION)
    Medical condition: Primary Mitochondrial Disease (PMD)
    Disease: Version SOC Term Classification Code Term Level
    22.0 10010331 - Congenital, familial and genetic disorders 10052641 Mitochondrial DNA mutation PT
    22.0 10010331 - Congenital, familial and genetic disorders 10052641 Mitochondrial DNA mutation PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) DE (Trial now transitioned)
    Trial results: (No results available)
    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: 2014-003346-27 Sponsor Protocol Number: ATYR1940-C-003 Start Date*: Information not available in EudraCT
    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 Early Onset and Other Pediatric Onset Fa...
    Medical condition: Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-001912-36 Sponsor Protocol Number: ATYR1940-C-005 Start Date*: 2015-08-04
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (F...
    Medical condition: 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 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    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: 2022-000389-16 Sponsor Protocol Number: 1821-FSH-301 Start Date*: 2022-08-08
    Sponsor Name:Fulcrum Therapeutics
    Full Title: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy
    Medical condition: Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) NL (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-002608-15 Sponsor Protocol Number: CBVS857X2202 Start Date*: 2014-06-12
    Sponsor Name:Novartis Farma SpA
    Full Title: A two-part placebo-controlled study to evaluate the safety, tolerability and preliminary efficacy of BVS857 in patients with spinal and bulbar muscular atrophy (SBMA)
    Medical condition: Spinal and bulbar muscular atrophy (SBMA)
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10068597 Bulbospinal muscular atrophy congenital PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: IT (Completed) DK (Completed) DE (Completed)
    Trial results: View results
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