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Clinical trials for Patient 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   44339   clinical trials with a EudraCT protocol, of which   7369   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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    29 result(s) found for: Patient advocacy. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2020-000980-21 Sponsor Protocol Number: PTC124-GD-048-DMD Start Date*: 2021-06-24
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy
    Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 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: 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: 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: 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: 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: 2022-000375-39 Sponsor Protocol Number: PTC743-CNS-005-LSEP Start Date*: 2022-12-07
    Sponsor Name:PTC THERAPEUTICS INC.
    Full Title: AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE
    Medical condition: Inherited mitochondrial disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) ES (Prematurely Ended) FR (Prematurely Ended) SE (Prematurely Ended) PL (Prematurely Ended)
    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: 2022-002668-65 Sponsor Protocol Number: PTC743-NEU-003e-FA Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
    Medical condition: Friedreich Ataxia (FA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    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: 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: 2016-001635-12 Sponsor Protocol Number: M15-562 Start Date*: 2017-11-23
    Sponsor Name:AbbVie Deutschland GmbH & Co. KG
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Multiple Dose Study to Assess Efficacy, Safety, Tolerability, and Pharmacokinetics of ABBV-8E12 in Progressive Supranuclear Palsy
    Medical condition: Progressive Supranuclear Palsy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10036813 Progressive supranuclear palsy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) FR (Completed) IT (Prematurely Ended) ES (Prematurely Ended)
    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: 2020-002812-36 Sponsor Protocol Number: PTC743-NEU-003-FA Start Date*: 2021-02-18
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA)
    Medical condition: Friedreich Ataxia (FA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Ongoing) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-003852-18 Sponsor Protocol Number: PTC518-CNS-002-HD Start Date*: 2022-06-02
    Sponsor Name:PTC Therapeutics, INC
    Full Title: A PHASE 2A, RANDOMIZED, PLACEBO-CONTROLLED, DOSE-RANGING STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PTC518 IN SUBJECTS WITH HUNTINGTON'S DISEASE
    Medical condition: Huntington’s disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) AT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-000474-29 Sponsor Protocol Number: PTC923-MD-003-PKU Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 Study of PTC923 in Subjects with Phenylketonuria
    Medical condition: Metabolic Disorders - Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Ongoing) PT (Completed) DK (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-000497-28 Sponsor Protocol Number: PTC923-MD-004-PKU Start Date*: Information not available in EudraCT
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
    Medical condition: Metabolic Disorders - Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) ES (Ongoing) PT (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2022-000073-12 Sponsor Protocol Number: PTC596-ONC-008-LMS Start Date*: 2022-06-22
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A PHASE 2/3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF UNESBULIN IN UNRESECTABLE OR METASTATIC, RELAPSED OR REFRACTORY LEIOMYOSARCOMA
    Medical condition: Leiomyosarcoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10024190 Leiomyosarcomas HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Prematurely Ended) ES (Prematurely Ended) IT (Completed) PL (Prematurely Ended) NL (Completed) FR (Completed)
    Trial results: (No results available)
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