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Clinical trials for Mitochondrial

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
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • 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   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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    127 result(s) found for: Mitochondrial. Displaying page 1 of 7.
    1  2  3  4  5  6  7  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: 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: 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: 2016-001696-79 Sponsor Protocol Number: KH176-201 Start Date*: 2016-09-06
    Sponsor Name:Khondrion BV
    Full Title: A double-blind, randomized, placebo-controlled, single-center, two-way cross-over study with KH176 in patients with the mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation and clinical signs of mitoc...
    Medical condition: Inherited mitochondrial disease, including MELAS (mitochondrial Encephalopathy Lactic Acidosis and Stroke like episodes) and MIDD (Maternally Inherited Diabetes and Deafness)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    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: 2012-002692-34 Sponsor Protocol Number: MitoFibrateCT1-2012 Start Date*: 2013-06-26
    Sponsor Name:Univ.-Klinik für Kinder- und Jugendheilkunde
    Full Title: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies
    Medical condition: mitochondrial disorder
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    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: 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: 2018-003000-39 Sponsor Protocol Number: 2018.0158 Start Date*: 2019-01-09
    Sponsor Name:St George’s University of London
    Full Title: A Multi-centre, Multiple-dose, Open-label Study to Investigate the Safety, Tolerability, Pharmacodynamics, and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP) in Patients with ...
    Medical condition: Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10058799 Mitochondrial encephalomyopathy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    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)
    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: 2021-002846-33 Sponsor Protocol Number: KL13332020-104A Start Date*: 2022-10-13
    Sponsor Name:Abliva AB
    Full Title: An interventional, randomised, double-blind, parallel-group, placebo-controlled, flexible-dose, adaptive study of the efficacy of KL1333 in adult patients with primary mitochondrial disease
    Medical condition: Adult patients with primary mitochondrial disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-002100-39 Sponsor Protocol Number: PTC743-MIT-001-EP Start Date*: 2021-07-26
    Sponsor Name:PTC THERAPEUTICS, INC.
    Full Title: Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)
    Medical condition: Genetically determined mitochondrial disease and associated refractory epilepsy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004852 10077953 Refractory epilepsy LLT
    20.0 100000004850 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Temporarily Halted) FR (Prematurely Ended) IT (Prematurely Ended) SE (Prematurely Ended) PL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-014263-40 Sponsor Protocol Number: MEL01 Start Date*: 2009-12-14
    Sponsor Name:Penwest Pharmaceuticals Co.
    Full Title: A Phase 2a, Double Blind, Randomized, Placebo-controlled, 28 day, Two-arm, Parallel Group Study of A0001 in Patients with the A3243G Mitochondrial DNA Point Mutation and Evidence of Impaired Mitoch...
    Medical condition: A3243G mitochondrial DNA point mutation and evidence of impaired mitochondrial function
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2022-000375-39 Sponsor Protocol Number: PTC743-CNS-005-LSEP Start Date*: 2022-12-07
    Sponsor Name:PTC THERAPEUTICS INC.
    Full Title: AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE
    Medical condition: Inherited mitochondrial disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) ES (Prematurely Ended) FR (Prematurely Ended) SE (Prematurely Ended) PL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2021-001268-58 Sponsor Protocol Number: 11500/AOGENO/2020 Start Date*: 2021-07-06
    Sponsor Name:Semmelweis University, STIA-POC-2020
    Full Title: Investigation of cellbiological effects of cariprazine in patients with mitochondrial schizophrenia
    Medical condition: Mitochondrial disease associated schizophrenia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10037175 - Psychiatric disorders 10039626 Schizophrenia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HU (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: 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: 2018-002721-29 Sponsor Protocol Number: 08486 Start Date*: 2018-12-24
    Sponsor Name:Newcastle upon Tyne Hospitals NHS Foundation Trust
    Full Title: Randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in patients with Mitochondrial Myopathy
    Medical condition: Mitochondrial Myopathy
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
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