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Clinical trials for Fabry and migalastat

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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    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44328   clinical trials with a EudraCT protocol, of which   7362   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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    14 result(s) found for: Fabry and migalastat. Displaying page 1 of 1.
    EudraCT Number: 2017-000146-21 Sponsor Protocol Number: AT1001-020 Start Date*: 2019-08-29
    Sponsor Name:Amicus Therapeutics, UK Ltd
    Full Title: AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABL...
    Medical condition: Fabry disease and with amenable GLA variants
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Ongoing) ES (Completed) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2019-000222-21 Sponsor Protocol Number: AT1001-036 Start Date*: 2020-08-25
    Sponsor Name:Amicus Therapeutics, UK Limited
    Full Title: A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTAT IN SUBJECTS > 12 YEARS OF AGE WITH FABRY DISEASE AND AMENABLE GLA VARIANTS
    Medical condition: Fabry disease and with amenable GLA variants
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2011-004800-40 Sponsor Protocol Number: AT1001-041 Start Date*: 2012-01-25
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease.
    Medical condition: Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A.
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) IT (Completed) DK (Completed) BE (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2007-001838-13 Sponsor Protocol Number: FAB-CL-205 Start Date*: 2009-05-05
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: Open Label Extension Study to Evaluate the Long-term Safety Tolerability and Pharmacodynamics of AT1001 in Patients with Fabry Disease
    Medical condition: Fabry Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2021-002320-20 Sponsor Protocol Number: EFC16158 Start Date*: 2022-04-11
    Sponsor Name:sanofi-aventis recherche et développement
    Full Title: A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry ...
    Medical condition: Fabry’s disease
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NO (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing) GR (Trial now transitioned) AT (Trial now transitioned) PL (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2005-004384-33 Sponsor Protocol Number: AA1565522 (FAB-CL-203) Start Date*: 2005-12-19
    Sponsor Name:Amicus Therapeutics
    Full Title: A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease
    Medical condition: Fabry Disease
    Disease: Version SOC Term Classification Code Term Level
    8.1 10016016 LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2006-000181-36 Sponsor Protocol Number: FAB-CL-204 Start Date*: 2006-03-17
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients with Fabry Disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    8.1 10016016 LLT
    Population Age: Adults Gender: Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-001133-40 Sponsor Protocol Number: AT1001-025 Start Date*: 2020-01-06
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment
    Medical condition: Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2014-002701-38 Sponsor Protocol Number: AT1001-042 Start Date*: 2014-12-17
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease
    Medical condition: Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A.
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) BE (Completed) GB (Completed) ES (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2010-022709-16 Sponsor Protocol Number: AT1001-013 Start Date*: 2011-09-12
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male
    Trial protocol: BE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-000065-20 Sponsor Protocol Number: LPS15919 Start Date*: 2020-02-05
    Sponsor Name:Sanofi Aventis Groupe (SAG)
    Full Title: A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing o...
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: NO (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-013459-31 Sponsor Protocol Number: AT1001-011 Start Date*: 2009-12-10
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS
    Medical condition: Fabry Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) FR (Completed) DE (Completed) NL (Completed) BE (Completed) ES (Completed) IT (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2010-022636-37 Sponsor Protocol Number: AT1001-012 Start Date*: 2011-01-18
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOU...
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) GB (Completed) DK (Completed) AT (Completed) GR (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-000667-24 Sponsor Protocol Number: ST-920-201 Start Date*: 2019-11-12
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Dise...
    Medical condition: Fabry Disease (X-linked lysosomal storage disease)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) IT (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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