Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Pompe 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   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    43 result(s) found for: Pompe Disease. Displaying page 2 of 3.
    « Previous 1  2  3  Next»
    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: 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: 2010-022231-11 Sponsor Protocol Number: AGLU07710 Start Date*: 2012-12-13
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age and Older
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    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: 2005-002759-42 Sponsor Protocol Number: AGLU02704 Start Date*: 2007-07-18
    Sponsor Name:Genzyme Europe BV
    Full Title: A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatmen...
    Medical condition: Glycogen Storage Disease type II (Pompe's disease)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036143 Pompe's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    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: 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: 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: 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: 2024-000461-24 Sponsor Protocol Number: LPS15677 Start Date*: 2024-12-18
    Sponsor Name:Genzyme
    Full Title: A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Late Onset Pompe Disease with One Year Alglucosidase Alfa Treatment
    Medical condition: Pompe's disease
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-004370-34 Sponsor Protocol Number: POM-003 Start Date*: 2018-04-09
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy
    Medical condition: Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) HU (Prematurely Ended)
    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: 2013-002257-30 Sponsor Protocol Number: AT2220-017 Start Date*: Information not available in EudraCT
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID α-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    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: 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
    EudraCT Number: 2021-000826-89 Sponsor Protocol Number: 35RC21_9909_PHARM-APO Start Date*: 2021-04-23
    Sponsor Name:CHU Rennes
    Full Title: Etude pharmacocinétique de l’apomorphine en perfusion sous-cutanée continue diurne chez le patient parkinsonien équilibré sous traitement
    Medical condition: Parkinson's disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10061536 Parkinson's disease PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000755-40 Sponsor Protocol Number: ATB200-03 Start Date*: 2019-04-08
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Algluc...
    Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) DK (Completed) GB (GB - no longer in EU/EEA) SE (Completed) HU (Completed) BE (Completed) SK (Completed) ES (Completed) NL (Completed) BG (Prematurely Ended) SI (Completed) GR (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-006045-10 Sponsor Protocol Number: loc/08-08VerinAPOTEP Start Date*: 2009-02-06
    Sponsor Name:CHU de Rennes
    Full Title: Effet de la stimulation dopaminergique continue par pompe à apomorphine sur les fluctuations cognitives et psychiques des patients parkinsoniens: étude clinique et métabolique cérébrale par TEP au ...
    Medical condition: L’objectif thérapeutique principal dans la maladie de Parkinson est de contrôler les fluctuations motrices. Cependant des fluctuations non motrices (FNM) sont fréquemment observées et semblent corr...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013113 Disease Parkinson's LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000954-67 Sponsor Protocol Number: ATB200-07 Start Date*: 2020-04-08
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
    Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Completed) DK (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) GR (Trial now transitioned) SI (Trial now transitioned) AT (Completed) IT (Trial now transitioned)
    Trial results: (No results available)
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    « Previous 1  2  3  Next»
    Query did not match any studies.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Tue May 06 09:54:04 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA