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Clinical trials for GM1

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: GM1. Displaying page 1 of 1.
    EudraCT Number: 2019-004949-32 Sponsor Protocol Number: P1-GM-101 Start Date*: 2021-03-30
    Sponsor Name:Lysogene
    Full Title: An open-label adaptive-design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis
    Medical condition: GM1 gangliosidosis
    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: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-001109-22 Sponsor Protocol Number: PBGM01-001 Start Date*: Information not available in EudraCT
    Sponsor Name:Passage Bio, Inc.
    Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Su...
    Medical condition: GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the human galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal).
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    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: (No results available)
    EudraCT Number: 2009-017026-39 Sponsor Protocol Number: UCBT-002 Start Date*: 2009-12-28
    Sponsor Name:Aldagen, Inc
    Full Title: A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM
    Medical condition: Inborn errors of metabolism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10062018 Inborn error of metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-003032-13 Sponsor Protocol Number: DF-07 Start Date*: 2016-09-15
    Sponsor Name:A.O.U. Università degli Studi della Campania "Luigi Vanvitelli"
    Full Title: A 16 week PILOT Study to determine the Frequency of Suphonylurea-Associated Hypoglycaemia in older moderately frail patients with type 2 diabetes mellitus poorly controlled on metformin: an open la...
    Medical condition: Type 2 Diabetes Mellitus
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004861 10012594 Diabetes LLT
    Population Age: Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-001863-10 Sponsor Protocol Number: CA001-050 Start Date*: 2021-04-12
    Sponsor Name:Bristol-Myers Squibb International Corporation
    Full Title: A Randomized, Open Label Phase II Clinical Trial of BMS-986012 in Combination with Carboplatin, Etoposide and Nivolumab as First-line Therapy in Extensive-Stage Small Cell Lung Cancer
    Medical condition: Extensive-stage Small Cell Lung Cancer
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10041068 Small cell lung cancer extensive stage PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PL (Trial now transitioned) NL (Trial now transitioned) BE (Trial now transitioned) GR (Trial now transitioned) IT (Trial now transitioned) RO (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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