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Clinical trials for Batten Disease

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   44335   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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    8 result(s) found for: Batten Disease. Displaying page 1 of 1.
    EudraCT Number: 2021-000706-21 Sponsor Protocol Number: AT-GTX-501-02 Start Date*: 2021-12-10
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE
    Medical condition: Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease.
    Disease: Version SOC Term Classification Code Term Level
    23.1 100000004850 10052074 Neuronal ceroid lipofuscinosis NOS LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-004623-23 Sponsor Protocol Number: AMO-02-MD-2-003 Start Date*: 2018-11-19
    Sponsor Name:AMO Pharma Ltd
    Full Title: A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy
    Medical condition: Treatment of child and adolescent congenital myotonic dystrophy.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2015-000891-85 Sponsor Protocol Number: 190-203 Start Date*: 2017-09-05
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease
    Medical condition: Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10052074 Neuronal ceroid lipofuscinosis NOS LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Completed)
    Trial results: View results
    EudraCT Number: 2012-005430-11 Sponsor Protocol Number: 190-201 Start Date*: 2013-08-15
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipo...
    Medical condition: Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2).
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10052074 Neuronal ceroid lipofuscinosis NOS LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2022-000889-18 Sponsor Protocol Number: DYNE101-DM1-201 Start Date*: 2023-02-08
    Sponsor Name:Dyne Therapeutics
    Full Title: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myoton...
    Medical condition: Myotonic Dystrophy Type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000067-16 Sponsor Protocol Number: AMO-02-MD-2-001 Start Date*: 2016-04-20
    Sponsor Name:AMO Pharma Ltd.
    Full Title: A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
    Medical condition: Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-000601-36 Sponsor Protocol Number: GUP15004 Start Date*: 2016-07-26
    Sponsor Name:FONDAZIONE SERENA ONLUS
    Full Title: CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1
    Medical condition: Myotonic Dystrophy type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-003480-37 Sponsor Protocol Number: 190-202 Start Date*: 2015-03-10
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease
    Medical condition: Late-Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10052074 Neuronal ceroid lipofuscinosis NOS LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) IT (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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