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Clinical trials for c9orf72

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   44364   clinical trials with a EudraCT protocol, of which   7388   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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    5 result(s) found for: c9orf72. Displaying page 1 of 1.
    EudraCT Number: 2021-002251-11 Sponsor Protocol Number: TPN-101-C9-201 Start Date*: 2021-12-06
    Sponsor Name:Transposon Therapeutics, Inc
    Full Title: A Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia)
    Medical condition: Amyotrophic lateral sclerosis or frontotemporal dementia due to a genetic mutation called C9orf72 hexanucleotide repeat expansion
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed) ES (Ongoing) BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-005193-94 Sponsor Protocol Number: WVE-004-001 Start Date*: 2021-12-20
    Sponsor Name:Wave Life Sciences UK Limited
    Full Title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Front...
    Medical condition: Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) NL (Completed) IE (Completed) SE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2022-002267-29 Sponsor Protocol Number: WVE-004-002 Start Date*: 2022-10-14
    Sponsor Name:Wave Life Sciences UK Limited
    Full Title: A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and...
    Medical condition: Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Prematurely Ended) IE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2019-000138-20 Sponsor Protocol Number: AL001-2 Start Date*: 2019-11-18
    Sponsor Name:Alector Inc.
    Full Title: A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9ORF72 Mutations Causative of...
    Medical condition: Frontotemporal Dementia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-002302-46 Sponsor Protocol Number: IMIB-TCIM/ELAII-2019-01 Start Date*: 2019-10-23
    Sponsor Name:Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
    Full Title: Phase II clinical trial of intramuscular infusion of autologous bone marrow stem cells in patients with amyotrophic lateral sclerosis
    Medical condition: Amyotrophic Lateral Sclerosis
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    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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