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Clinical trials for Health advocacy

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   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    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.
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    14 result(s) found for: Health advocacy. Displaying page 1 of 1.
    EudraCT Number: 2021-000706-21 Sponsor Protocol Number: AT-GTX-501-02 Start Date*: 2021-12-10
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE
    Medical condition: Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease.
    Disease: Version SOC Term Classification Code Term Level
    23.1 100000004850 10052074 Neuronal ceroid lipofuscinosis NOS LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    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)
    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: 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: 2020-002100-39 Sponsor Protocol Number: PTC743-MIT-001-EP Start Date*: 2021-07-26
    Sponsor Name:PTC THERAPEUTICS, INC.
    Full Title: Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)
    Medical condition: Genetically determined mitochondrial disease and associated refractory epilepsy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004852 10077953 Refractory epilepsy LLT
    20.0 100000004850 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Temporarily Halted) FR (Prematurely Ended) IT (Prematurely Ended) SE (Prematurely Ended) PL (Prematurely Ended)
    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: 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: 2022-001013-39 Sponsor Protocol Number: PTC124-GD-028-ANI Start Date*: 2022-04-14
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia
    Medical condition: Nonsense Mutation Aniridia
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-001872-13 Sponsor Protocol Number: PTC299-VIR-015-COV19 Start Date*: 2021-01-15
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Evaluation of the efficacy and safety of PTC299 in hospitalized subjects with COVID-19 (FITE19)
    Medical condition: Coronavirus disease 2019 (COVID-19)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004862 10066740 Acute respiratory tract infection LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Prematurely Ended) FR (Completed) BE (Completed) GR (Prematurely Ended)
    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: 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: 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: 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
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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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