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Clinical trials for Glycogen storage disease type I

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
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    • 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   44336   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: Glycogen storage disease type I. Displaying page 1 of 1.
    EudraCT Number: 2018-004473-27 Sponsor Protocol Number: 401GSDIA02 Start Date*: 2021-08-25
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen St...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-005449-12 Sponsor Protocol Number: FARM5S3JT5 Start Date*: 2006-10-11
    Sponsor Name:Dipartimento di Pediatria
    Full Title: EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b
    Medical condition: As concern objective number 1: Patients affected by glycogen storage disease (GSD) type 1a and patients affected by GSD1b with renal dysfunction including glomerular hyperfiltration, microalbuminu...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018464 Glycogen storage disease type I LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004184-12 Sponsor Protocol Number: DTX401-CL301 Start Date*: 2022-08-22
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia....
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) ES (Trial now transitioned) IT (Trial now transitioned) DK (Trial now transitioned) PT (Completed) DE (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-003023-30 Sponsor Protocol Number: 401GSDIA01 Start Date*: 2018-11-27
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Dise...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2020-004686-39 Sponsor Protocol Number: EFC14462 Start Date*: Information not available in EudraCT
    Sponsor Name:Sanofi-Aventis Recherche & Développement
    Full Title: An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participan...
    Medical condition: Glycogen storage disease type II
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) Outside EU/EEA ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-001805-28 Sponsor Protocol Number: POM-002 Start Date*: 2012-07-03
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2010-023561-22 Sponsor Protocol Number: POM-001 Start Date*: 2011-08-15
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe...
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2021-000580-78 Sponsor Protocol Number: EMPAtia Start Date*: 2022-02-02
    Sponsor Name:The Children’s Memorial Health Institute
    Full Title: "The assessment of effectiveness and safeness of utilizing empagliflozin in the treatment of neutropenia of patients suffering from glycogenolysis type Ib"
    Medical condition: neutropenia and / or abnormal neutrophil function in glycogenosis I b
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: PL (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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