Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Fabry

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   44335   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
     
    42 result(s) found for: Fabry. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2023-000624-11 Sponsor Protocol Number: LPS16583 Start Date*: 2023-06-21
    Sponsor Name:Genzyme Europe B.V.
    Full Title: A Phase 4, open label, safety and efficacy study of Fabrazyme® (agalsidase beta) as enzyme replacement therapy in Chinese participants with Fabry Disease
    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: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 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: 2021-002350-90 Sponsor Protocol Number: EFC17045 Start Date*: 2022-04-01
    Sponsor Name:Sanofi-Aventis Recherche & Développement
    Full Title: A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female participants ≥16 Years of Age with F...
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Trial now transitioned) DE (Ongoing) DK (Trial now transitioned) AT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) NO (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2004-000772-14 Sponsor Protocol Number: TKT027 Start Date*: 2004-07-23
    Sponsor Name:TKT Inc
    Full Title: A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027)
    Medical condition: Fabry Disease
    Disease: Version SOC Term Classification Code Term Level
    6.1 10016016 PT
    Population Age: Adults Gender: Male
    Trial protocol: CZ (Completed)
    Trial results: Removed from public view
    EudraCT Number: 2015-000697-35 Sponsor Protocol Number: AGAL19412 Start Date*: 2015-04-24
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study)
    Medical condition: Fabry Disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    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: 2016-001318-11 Sponsor Protocol Number: PB-102-F30 Start Date*: 2017-03-24
    Sponsor Name:Protalix Ltd.
    Full Title: An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)
    Medical condition: Fabry disease (α-galactosidase A deficiency)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) CZ (Completed) DE (Completed) SI (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2009-015985-75 Sponsor Protocol Number: HGT-REP-060 Start Date*: 2010-02-03
    Sponsor Name:Shire Human Genetic Therapies (HGT), Inc.
    Full Title: An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PL (Completed) CZ (Completed) GB (Completed) FI (Completed) SI (Completed)
    Trial results: View results
    EudraCT Number: 2018-004689-32 Sponsor Protocol Number: SHP675-301 Start Date*: 2021-09-24
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease
    Medical condition: Fabry 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, Elderly Gender: Male, Female
    Trial protocol: PT (Prematurely Ended) FI (Prematurely Ended) GR (Prematurely Ended) PL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-003369-85 Sponsor Protocol Number: ID-069A301 Start Date*: 2018-05-14
    Sponsor Name:Idorsia Pharmaceuticals Ltd
    Full Title: A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
    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: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) AT (Completed) NL (Completed) BE (Completed) IE (Completed) ES (Ongoing) NO (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-001148-67 Sponsor Protocol Number: PB-102-F60 Start Date*: 2018-09-26
    Sponsor Name:Protalix Ltd.
    Full Title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
    Medical condition:
    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, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NO (Completed) CZ (Completed) ES (Ongoing) SI (Completed) NL (Completed) HU (Completed) IT (Completed) FI (Completed) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-004995-49 Sponsor Protocol Number: LTS14116 Start Date*: 2015-10-14
    Sponsor Name:Genzyme Corporation
    Full Title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed with Fabry Disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (Completed) PL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2018-002097-51 Sponsor Protocol Number: FLT190-01 Start Date*: 2020-02-12
    Sponsor Name:Freeline Therapeutics Ltd
    Full Title: A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease
    Medical condition: Fabry 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, Elderly Gender: Male
    Trial protocol: DE (Prematurely Ended) DK (Prematurely Ended) FR (Prematurely Ended) IT (Prematurely Ended) AT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-004786-40 Sponsor Protocol Number: PB-102-F01 Start Date*: 2013-05-10
    Sponsor Name:Protalix Ltd.
    Full Title: A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks ...
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2012-001966-14 Sponsor Protocol Number: AGAL19110 Start Date*: 2012-10-25
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients with Fabry Disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    15.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2007-005668-28 Sponsor Protocol Number: AGAL06207 Start Date*: 2008-07-29
    Sponsor Name:Genzyme Europe BV
    Full Title: A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symp...
    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: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (Completed) NL (Completed) PT (Prematurely Ended) DE (Prematurely Ended) CZ (Completed) FR (Completed)
    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: 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
    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.
    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-Fri May 09 23:03:49 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA