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Clinical trials for Charcot-Marie-Tooth disease

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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   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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    11 result(s) found for: Charcot-Marie-Tooth disease. Displaying page 1 of 1.
    EudraCT Number: 2015-001716-36 Sponsor Protocol Number: 6100 Start Date*: 2015-08-03
    Sponsor Name:HÔPITAUX UNIVERSITAIRES DE STRASBOURG
    Full Title: LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)
    Medical condition: Charcot-Marie-Tooth disease type 1A
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adults Gender: Male
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-023097-40 Sponsor Protocol Number: CLN-PXT3003-01 Start Date*: 2010-11-29
    Sponsor Name:Pharnext
    Full Title: A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A
    Medical condition: Charcot-Marie-Tooth disease (type 1A).
    Disease: Version SOC Term Classification Code Term Level
    12.1 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004805-30 Sponsor Protocol Number: CLN-PXT3003-06 Start Date*: Information not available in EudraCT
    Sponsor Name:Pharnext SCA
    Full Title: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)
    Medical condition: Charcot Marie Tooth Type 1A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) DK (Prematurely Ended) ES (Prematurely Ended) IT (Prematurely Ended) FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-002378-19 Sponsor Protocol Number: CLN-PXT3003-02 Start Date*: 2015-08-27
    Sponsor Name:PHARNEXT
    Full Title: International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-T...
    Medical condition: Charcot-Marie-Tooth Disease - Type 1A
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) DE (Suspended by CA) ES (Completed) BE (Completed) GB (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-001150-15 Sponsor Protocol Number: MD1003CT2015-01-SERENDEM Start Date*: 2016-03-14
    Sponsor Name:MEDDAY PHARMACEUTICALS
    Full Title: SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study
    Medical condition: chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating neuropathy associated with immunoglobulin M (IgM) monoclonal gammopathy and antibodies against myelin-associated glycoprotei...
    Disease: Version SOC Term Classification Code Term Level
    18.1 10029205 - Nervous system disorders 10066137 Anti-MAG neuropathy LLT
    18.1 10029205 - Nervous system disorders 10057645 Chronic inflammatory demyelinating polyradiculoneuropathy PT
    18.1 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2015-002379-81 Sponsor Protocol Number: CLN-PXT3003-03 Start Date*: 2017-01-26
    Sponsor Name:Pharnext
    Full Title: International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.
    Medical condition: Charcot-Marie-Tooth Disease type 1A
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) DE (Suspended by CA) BE (Completed) NL (Completed) ES (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-000305-79 Sponsor Protocol Number: A083-04 Start Date*: 2019-05-14
    Sponsor Name:Acceleron Pharma Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with C...
    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: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002827-14 Sponsor Protocol Number: TreatSPG11 Start Date*: 2021-01-19
    Sponsor Name:IRCCS Fondazione Stella Maris
    Full Title: Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11
    Medical condition: Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy o...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10019903 Hereditary spastic paraplegia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-000032-27 Sponsor Protocol Number: GUP05007 Start Date*: 2005-10-19
    Sponsor Name:ISTITUTO NEUROLOGICO CARLO BESTA
    Full Title: MULTICENTRE RANDOMISED DOUBLE BLIND PLACEBO CONTROLLED TRIAL OF LONG-TERM ASCORBIC ACID TREATMENT IN CHARCOT-MARIE-TOOTH DISEASE TYPE 1A CMT-TRIAAL CMT-TRial Italian with Ascorbic Acid Long term
    Medical condition: Charcot-Marie-Tooth disease type 1A CMT1A
    Disease: Version SOC Term Classification Code Term Level
    6.1 10034606 HLGT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-012073-34 Sponsor Protocol Number: R0000063 Start Date*: 2009-07-08
    Sponsor Name:Radboud University Nijmegen Medical Centre
    Full Title: Botulinum toxin for treating calf muscle spasticity in Hereditary Spastic Paraparesis (HSP): functional effects on dynamic balance and gait
    Medical condition: Subjects with hereditary spastic paraparesis (HSP)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10019903 Hereditary spastic paraplegia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2022-000491-18 Sponsor Protocol Number: AT-007-1005 Start Date*: 2022-09-14
    Sponsor Name:Applied Therapeutics Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Two-Part Study to Evaluate the Pharmacodynamic Efficacy and Clinical Benefit of AT 007 in Patients with Sorbitol Dehydrogenase (SORD) Deficiency
    Medical condition: SoRbitol Dehydrogenase (SORD) DEficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004852 10029328 Neuropathy LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: CZ (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
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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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