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Clinical trials for Glycogen Storage Disease Type II

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    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).

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    34 result(s) found for: Glycogen Storage Disease Type II. Displaying page 2 of 2.
    « Previous 1  2 
    EudraCT Number: 2015-000583-34 Sponsor Protocol Number: AGLU03707_MSC12817 Start Date*: 2015-04-15
    Sponsor Name:Genzyme Corporation Inc
    Full Title: An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients with Pompe Disease Who Have Previously Received Myozyme
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-000584-14 Sponsor Protocol Number: AGLU03807,MSC12862 Start Date*: 2015-04-15
    Sponsor Name:Genzyme Corporation Inc
    Full Title: An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naïve CRIM(-) Patients with Infantile-Onset Pompe Disease
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-005595-42 Sponsor Protocol Number: AGLU07510 Start Date*: 2013-04-17
    Sponsor Name:Genzyme Corporation, a Sanofi company
    Full Title: A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients with Infantile-Onset Pompe Di...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2011-002154-32 Sponsor Protocol Number: AT2220-010 Start Date*: 2011-12-07
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE
    Medical condition: Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (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: 2016-000942-77 Sponsor Protocol Number: EFC14028 Start Date*: 2016-07-15
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase ...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DK (Prematurely Ended) SE (Prematurely Ended) NL (Completed) ES (Completed) CZ (Completed) DE (Completed) BE (Completed) AT (Completed) PL (Completed) IT (Completed) PT (Completed) BG (Completed) HU (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-002933-12 Sponsor Protocol Number: 11/0090 Start Date*: 2014-04-07
    Sponsor Name:University College London,
    Full Title: A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdom...
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-001283-30 Sponsor Protocol Number: SPK-3006-101 Start Date*: 2020-06-16
    Sponsor Name:Spark Therapeutics
    Full Title: Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease
    Medical condition: Pompe Disease (also known as 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: Adults Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) DE (Trial now transitioned) NL (Ongoing) DK (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-000582-31 Sponsor Protocol Number: AGLU3306 Start Date*: 2015-04-15
    Sponsor Name:Genzyme Corporation Inc
    Full Title: An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal R...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-004798-34 Sponsor Protocol Number: ATB200-02 Start Date*: 2016-08-01
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINI...
    Medical condition: Pompe Disease - acid maltase deficiency or glycogen storage disease type II.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-002829-31 Sponsor Protocol Number: AGLU02804, Amendment 1 Start Date*: 2005-08-31
    Sponsor Name:Genzyme Europe BV
    Full Title: Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Lat...
    Medical condition: Glycogen Storage Disease type II (Pompe´s disease)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2007-001375-11 Sponsor Protocol Number: not applicable Start Date*: 2007-05-16
    Sponsor Name:
    Full Title: Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme
    Medical condition: Pompe disease (glycogen storage disease type II) is a genetic, lysosomal storage disorder with a frequency of 1 in 40.000 newborns. The disease is caused by deficiency of alpha-glucosidase, a lysos...
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2005-001629-27 Sponsor Protocol Number: AGLU02403 Start Date*: 2005-08-03
    Sponsor Name:Genzyme Europe BV
    Full Title: A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602
    Medical condition: Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glyco...
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-004578-16 Sponsor Protocol Number: VAL-1221-201-16 Start Date*: 2017-06-19
    Sponsor Name:Valerion Therapeutics, LLC
    Full Title: A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patien...
    Medical condition: Late-Onset GSD-II (Pompe Disease)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10036143 Pompe's disease LLT
    20.1 100000004850 10045253 Type II glycogen storage disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
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