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Clinical trials for Leigh syndrome

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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    6 result(s) found for: Leigh syndrome. Displaying page 1 of 1.
    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: 2012-001294-84 Sponsor Protocol Number: OPBGC&RS2011-004_CA2012 Start Date*: 2012-09-03
    Sponsor Name:EDISON PHARMACEUTICALS INC.
    Full Title: Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease
    Medical condition: Leigh Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    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: 2006-006886-18 Sponsor Protocol Number: ALK21-014 Start Date*: 2007-06-04
    Sponsor Name:Alkermes Inc
    Full Title: Efficacy and Safety of Vivitrol® in Adults Completing Inpatient Treatment for Alcohol Dependence
    Medical condition: Alcohol Dependence
    Disease: Version SOC Term Classification Code Term Level
    9.1 10001590 Alcohol addiction LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) AT (Completed)
    Trial results: View results
    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: 2020-003232-24 Sponsor Protocol Number: AVA-PED-301 Start Date*: 2020-12-03
    Sponsor Name:Dova Pharmaceuticals, Inc.
    Full Title: A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment...
    Medical condition: Thrombocytopenia in paediatric subjects with immune thrombocytopenia for ≥6 months duration who have had an insufficient response to a previous treatment
    Disease: Version SOC Term Classification Code Term Level
    20.0 10005329 - Blood and lymphatic system disorders 10043555 Thrombocytopenias HLT
    23.0 10005329 - Blood and lymphatic system disorders 10083843 Primary immune thrombocytopenia LLT
    23.0 10005329 - Blood and lymphatic system disorders 10083842 Immune thrombocytopenia PT
    20.0 10005329 - Blood and lymphatic system disorders 10035534 Platelet disorders HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) HU (Completed) DE (Completed) PL (Completed)
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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