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Clinical trials

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   44009   clinical trials with a EudraCT protocol, of which   7316   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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    2,070 result(s) found. Displaying page 32 of 104.
    EudraCT Number: 2021-004253-22 Sponsor Protocol Number: ALXN2040-PNH-303 Start Date*: 2022-07-04
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglo...
    Medical condition: Patients with Paroxysmal Nocturnal Hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Trial now transitioned) IT (Ongoing) GR (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-001715-79 Sponsor Protocol Number: BL-8040.SCM.301 Start Date*: 2019-05-14
    Sponsor Name:BioLineRx Ltd.
    Full Title: A Phase III, Randomized, Double-Blinded, Placebo-Controlled, Multi-Centre Study Evaluating the Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and G-CSF as compared to Placebo...
    Medical condition: Hematopoietic Stem Cell mobilization in Multiple Myeloma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10028228 Multiple myeloma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Trial now transitioned) CZ (Prematurely Ended) ES (Ongoing) DE (Trial now transitioned) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-002611-26 Sponsor Protocol Number: AVXS-101-LT-002 Start Date*: 2020-03-30
    Sponsor Name:AveXis, Inc.
    Full Title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Trial now transitioned) ES (Prematurely Ended) FR (Trial now transitioned) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004494-41 Sponsor Protocol Number: CLIN2001UCM301 Start Date*: 2021-06-11
    Sponsor Name:Steba Biotech, S.A.
    Full Title: Multicenter Phase 3 Pivotal Study to Evaluate the Safety and Efficacy of TOOKAD (padeliporfin) Vascular Targeted Photodynamic Therapy in the Treatment of Low Grade Upper Tract Urothelial Cancer
    Medical condition: Low Grade Upper Tract Urothelial Cancer
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10077840 Urothelial cancer of renal pelvis LLT
    20.0 100000004864 10046375 Ureter cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) IT (Ongoing) AT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-003333-11 Sponsor Protocol Number: AG348-C-023 Start Date*: 2022-04-25
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regular...
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) FR (Trial now transitioned) IT (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-006057-21 Sponsor Protocol Number: CABL001A2302 Start Date*: 2021-09-29
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase 3b, multi-center, open-label, treatment optimization study of oral asciminib in patients with Chronic Myelogenous Leukemia in chronic phase (CML-CP) previously treated with 2 or more tyrosi...
    Medical condition: Chronic Myelogenous Leukemia in chronic phase (CML-CP)
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10009012 Chronic myelogenous leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Trial now transitioned) ES (Ongoing) DE (Trial now transitioned) IT (Ongoing) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-004561-13 Sponsor Protocol Number: ISIS420915-CS3 Start Date*: 2014-07-04
    Sponsor Name:Ionis Pharmaceuticals, Inc
    Full Title: An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)
    Medical condition: Familial Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed) GB (GB - no longer in EU/EEA) DE (Completed) FR (Completed) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2021-000211-23 Sponsor Protocol Number: AG348-C-017 Start Date*: 2021-10-15
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non Transfusion-Dependent Alpha- or Beta-Thalassemia (ENE...
    Medical condition: Non–Transfusion-Dependent Alpha- or Beta-Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) DK (Trial now transitioned) IT (Ongoing) BG (Trial now transitioned) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-000212-34 Sponsor Protocol Number: AG348-C-018 Start Date*: 2021-10-11
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZ...
    Medical condition: Transfusion-Dependent Alpha- or Beta-Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    22.0 100000004850 10081904 Transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) DK (Trial now transitioned) IT (Ongoing) BG (Trial now transitioned) NL (Ongoing)
    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 (Ongoing) PL (Prematurely Ended) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-000007-21 Sponsor Protocol Number: 212620 Start Date*: 2021-08-25
    Sponsor Name:GlaxoSmithKline Research & Development Limited
    Full Title: A two-part, randomized, placebo controlled, double blind, multicenter, Phase 3 study to evaluate the efficacy and safety of linerixibat for the treatment of cholestatic pruritus in participants wit...
    Medical condition: Moderate to severe Cholestatic pruritus with primary biliary cholangitis (PBC).
    Disease: Version SOC Term Classification Code Term Level
    24.1 10040785 - Skin and subcutaneous tissue disorders 10064190 Cholestatic pruritus PT
    21.0 10019805 - Hepatobiliary disorders 10080429 Primary biliary cholangitis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) IT (Ongoing) CZ (Completed) GR (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-001565-33 Sponsor Protocol Number: RA101495-02.302 Start Date*: 2020-04-24
    Sponsor Name:Ra Pharmaceuticals, Inc.
    Full Title: A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
    Medical condition: Generalized Myasthenia Gravis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10028395 - Musculoskeletal and connective tissue disorders 10028415 Myasthenia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NO (Trial now transitioned) GB (GB - no longer in EU/EEA) ES (Ongoing) IT (Ongoing) PL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-001831-17 Sponsor Protocol Number: MT-7117-A-301 Start Date*: 2022-04-07
    Sponsor Name:Mitsubishi Tanabe Pharma Development America (MTDA), Inc.
    Full Title: A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Pr...
    Medical condition: Erythropoietic Protoporphyria or X-Linked Protoporphyria
    Disease: Version SOC Term Classification Code Term Level
    24.0 100000004850 10015289 Erythropoietic protoporphyria LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Ongoing) ES (Ongoing) SE (Trial now transitioned) NO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-003467-26 Sponsor Protocol Number: NN7769-4516 Start Date*: 2021-11-30
    Sponsor Name:Novo Nordisk A/S
    Full Title: Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors
    Medical condition: Haemophilia A Haemophilia A with inhibitors
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018938 Haemophilia A (Factor VIII) LLT
    20.0 100000004850 10053751 Hemophilia A with anti factor VIII LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed) PL (Ongoing) IT (Ongoing) ES (Ongoing) PT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-004740-24 Sponsor Protocol Number: INCB54828-209 Start Date*: 2022-06-29
    Sponsor Name:Incyte Corporation
    Full Title: A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System ...
    Medical condition: Participants at least 18 years of age who have recurrent GBM or other recurrent gliomas, circumscribed astrocytic gliomas, and glioneuronal and neuronal tumors, harboring an FGFR1-3 mutation or fus...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065147 Malignant solid tumor LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing) ES (Ongoing) DK (Ongoing) IT (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Ongoing) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-001498-21 Sponsor Protocol Number: A011-14 Start Date*: 2021-11-11
    Sponsor Name:Acceleron Pharma Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sotatercept When Added to Maximum Tolerated Background Therapy in Participants With Pulmonary Arterial Hypertension (PAH) W...
    Medical condition: Pulmonary Arterial Hypertension (PAH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10064911 Pulmonary arterial hypertension PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) IT (Ongoing) NL (Ongoing) BE (Trial now transitioned) DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-003374-91 Sponsor Protocol Number: SRP-9001-301 Start Date*: 2022-09-12
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: FR (Ongoing) BE (Ongoing) ES (Ongoing) DE (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-005881-17 Sponsor Protocol Number: RIN-PF-303 Start Date*: 2022-08-18
    Sponsor Name:UNITED THERAPEUTICS CORPORATION
    Full Title: A Randomized, Double-blind, Placebo-controlled, Multinational, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects with Idiopathic Pulmonary Fibrosis (TETON-2)
    Medical condition: Idiopathic Pulmonary Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10021240 Idiopathic pulmonary fibrosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) DK (Trial now transitioned) FR (Trial now transitioned) IT (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-023691-33 Sponsor Protocol Number: MUV-MEMMAT-01 Start Date*: 2011-05-05
    Sponsor Name:Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
    Full Title: A Phase II study of metronomic and targeted anti-angiogenesis therapy for children with recurrent/progressive medulloblastoma, ependymoma and ATRT
    Medical condition: Recurrent/progressive medulloblastoma, ependymoma and ATRT
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Ongoing) SE (Restarted) CZ (Trial now transitioned) DK (Ongoing) ES (Ongoing)
    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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