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Clinical trials for ataxia

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   44352   clinical trials with a EudraCT protocol, of which   7379   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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    20 result(s) found for: ataxia. Displaying page 1 of 1.
    EudraCT Number: 2011-004104-38 Sponsor Protocol Number: ARPA-IGF1-2011 Start Date*: 2012-02-17
    Sponsor Name:FIBHULP
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-004001-96 Sponsor Protocol Number: ATFD07 Start Date*: 2007-12-20
    Sponsor Name:Hospital Sant Joan de Déu
    Full Title: Efectos del tratamiento con deferiprone sobre la hipertrofia miocárdica y los parámetros de función cardiaca en pacientes afectos de Ataxia de Friedreich
    Medical condition: La Ataxia de Friedreich es una enfermedad causada por un déficit de la proteína frataxina. Su déficit produce acúmulo de hierro libre intramitocondrial, un incremento de la producción de radicales ...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10017374 Friedreich's ataxia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-007653-31 Sponsor Protocol Number: EC07/90471 Start Date*: 2008-06-12
    Sponsor Name:Fundación Investigación Biomédica Hospital La Paz
    Full Title: Estudio aleatorizado, doble ciego, controlado con placebo, de 6 meses de duración para evaluar la eficacia de eritropoyetina recombinante humana en pacientes con ataxia de Friedreich. Randomized, d...
    Medical condition: Ataxia de Friedreich Friedreich´s ataxia CIE : 334,0
    Disease: Version SOC Term Classification Code Term Level
    9 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-019737-94 Sponsor Protocol Number: ARPA-IGF1-2010 Start Date*: 2010-07-13
    Sponsor Name:FIBHULP
    Full Title: ESTUDIO ABIERTO, DE EFICACIA, TOLERANCIA Y SEGURIDAD DEL TRATAMIENTO CON EL FACTOR TRÓFICO IGF-I EN LA ATAXIA CEREBELOSA DEGENERATIVA
    Medical condition: Ataxia cerebelosa degenerativa de grado leve a moderado
    Disease: Version SOC Term Classification Code Term Level
    11 10008025 Ataxia cerebelosa LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-002812-36 Sponsor Protocol Number: PTC743-NEU-003-FA Start Date*: 2021-02-18
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA)
    Medical condition: Friedreich Ataxia (FA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Ongoing) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2022-002668-65 Sponsor Protocol Number: PTC743-NEU-003e-FA Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
    Medical condition: Friedreich Ataxia (FA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-010865-22 Sponsor Protocol Number: LA29-EXT Start Date*: 2009-06-30
    Sponsor Name:ApoPharma Incorporated
    Full Title: An open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich's ataxia.
    Medical condition: Friedreich's ataxia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10017374 Friedreich's ataxia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) IT (Completed) BE (Completed) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-002163-17 Sponsor Protocol Number: 15-138 Start Date*: 2018-10-25
    Sponsor Name:RWTH Aachen University represented by the Center for Translational & Clinical Research Aachen (CTC-A)
    Full Title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA.
    Medical condition: Friedreich Ataxia
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) AT (Prematurely Ended) GB (GB - no longer in EU/EEA) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2018-000338-36 Sponsor Protocol Number: IEDAT-03-2018 Start Date*: 2018-05-31
    Sponsor Name:EryDel S.p.A.
    Full Title: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
    Medical condition: Patient with neurological symptoms of Ataxia Telangiectasia
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10003594 Ataxia telangiectasia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed) DE (Completed) BE (Completed) PL (Completed) GB (GB - no longer in EU/EEA) NO (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2022-001314-19 Sponsor Protocol Number: VO659-CT01 Start Date*: 2023-04-25
    Sponsor Name:VICO Therapeutics B.V.
    Full Title: A phase 1/2a, open-label trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of intrathecally administered VO659 in participants with sp...
    Medical condition: spinocerebellar ataxia types 1, 3 and Huntington’s disease
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10057660 Spinocerebellar ataxia LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) NL (Trial now transitioned) IT (Prematurely Ended) DK (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-001605-24 Sponsor Protocol Number: GWND19002 Start Date*: 2020-01-15
    Sponsor Name:GW Research Ltd
    Full Title: An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome
    Medical condition: Rett syndrome (RTT) [typical or atypical]
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10077709 Rett syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Prematurely Ended) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2018-004331-71 Sponsor Protocol Number: IB1001-201 Start Date*: 2019-05-08
    Sponsor Name:IntraBio Ltd
    Full Title: Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study
    Medical condition: Niemann-Pick Disease type C (NPC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) ES (Ongoing) SK (Completed)
    Trial results: View results
    EudraCT Number: 2018-004406-25 Sponsor Protocol Number: IB1001-202 Start Date*: 2019-05-09
    Sponsor Name:IntraBio Ltd
    Full Title: Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study.
    Medical condition: GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2020-003136-25 Sponsor Protocol Number: CTD-TCNPC-301 Start Date*: 2021-10-25
    Sponsor Name:Cyclo Therapeutics, Inc.
    Full Title: A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl β cycl...
    Medical condition: Niemann Pick Disease Type C1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) DE (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-004058-11 Sponsor Protocol Number: RM-493-023 Start Date*: Information not available in EudraCT
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström syndrome (AS) Patients with Moderate to Severe Obesity
    Medical condition: Obesity and hyperphagia in patients with Bardet-Biedl Syndrome or Alström syndrome
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) ES (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2011-000990-29 Sponsor Protocol Number: IC2011-02 Start Date*: Information not available in EudraCT
    Sponsor Name:INSTITUT CURIE
    Full Title: Multinational européan trial for children with the opsoclonus myoclonus syndrome/dancing eye syndrome.
    Medical condition: Opsoclonus myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in children with and without neuroblastoma (NBpos and NBneg)
    Disease: Version SOC Term Classification Code Term Level
    15.0 10015919 - Eye disorders 10053854 Opsoclonus myoclonus PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) SE (Completed) IT (Prematurely Ended) GB (GB - no longer in EU/EEA) AT (Completed) ES (Ongoing) BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-004438-93 Sponsor Protocol Number: CT-ORZY-NPC-002 Start Date*: 2016-05-09
    Sponsor Name:Orphazyme A/S
    Full Title: Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C
    Medical condition: Niemann Pick disease type C
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) DE (Completed) IT (Completed) PL (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA)
    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: 2018-003291-12 Sponsor Protocol Number: SHP611-201 Start Date*: 2019-04-11
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
    Medical condition: Late Metachromatic Leukodystrophy (MLD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) NL (Completed) ES (Trial now transitioned) BE (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) IT (Trial now transitioned) GR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-002375-34 Sponsor Protocol Number: EFC15299 Start Date*: 2020-07-23
    Sponsor Name:Genzyme Corporation
    Full Title: A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2...
    Medical condition: Tay-Sachs disease Sandhoff disease
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10043147 Tay-Sachs disease LLT
    23.0 10010331 - Congenital, familial and genetic disorders 10081314 Sandhoff disease LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) GB (GB - no longer in EU/EEA) PT (Prematurely Ended) AT (Completed) FR (Completed) ES (Completed) CZ (Prematurely Ended) IT (Completed)
    Trial results: View results
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