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Clinical trials for Genetic diseases

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   44338   clinical trials with a EudraCT protocol, of which   7368   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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    2,477 result(s) found for: Genetic diseases. Displaying page 1 of 124.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2018-000029-29 Sponsor Protocol Number: 95105003 Start Date*: 2018-12-11
    Sponsor Name:VU University Medical Center
    Full Title: Extra energy for hearts with a genetic defect: ENERGY trial
    Medical condition: Hypertrophic cardiomyopathy
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-001888-39 Sponsor Protocol Number: PGX-HT Start Date*: 2016-01-28
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: PHARMACOGENOMICS HYPERTENSION : A NEW APPROACH FOR PERSONALIZED MEDICINE ( PGX - HT )
    Medical condition: Hypertension
    Disease: Version SOC Term Classification Code Term Level
    19.0 10047065 - Vascular disorders 10051753 Vascular calcification PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-003938-32 Sponsor Protocol Number: 11190801 Start Date*: 2014-02-12
    Sponsor Name:Medizinische Universität Graz
    Full Title: Genetic alterations in genes coding for transporters and metformin resistance
    Medical condition: We want to investigate the pharmacokintetics of metformin in patients with type 2 diabetes or polycystic ovary syndrome in association with the patients genotype
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed)
    Trial results: View results
    EudraCT Number: 2013-002124-17 Sponsor Protocol Number: IMO-AFLI-2013-01 Start Date*: 2013-10-04
    Sponsor Name:Fundació de Recerca de L?Institut de Microcirurgia Ocular
    Full Title: Phase IV study to evaluate genetic variants of VEGF pathway as biomarkers of VEGF Trap-Eye treatment efficacy in subjects with neovascular age-related macular degeneration (wAMD).
    Medical condition: Wet age-related macular degeneration
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2005-004008-36 Sponsor Protocol Number: ANAK-01 Start Date*: 2005-10-27
    Sponsor Name:ISTITUTO GIANNINA GASLINI
    Full Title: Efficacy and safety of treatment with Anakinra in patients with CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Medical condition: CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-002319-86 Sponsor Protocol Number: DIAN-Obs-BCN Start Date*: 2019-03-20
    Sponsor Name:Fundació Clínic per a la Recerca Biomèdica
    Full Title: Dominantly Inherited Alzheimer Network - DIAN
    Medical condition: Alzheimer disease
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005032-30 Sponsor Protocol Number: FLT201-01 Start Date*: 2023-03-13
    Sponsor Name:Freeline Therapeutics Limited
    Full Title: A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1)
    Medical condition: Type 1 Gaucher Disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-001118-32 Sponsor Protocol Number: EP06-401 Start Date*: 2011-05-25
    Sponsor Name:Sandoz GmbH
    Full Title: Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia
    Medical condition: Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10005329 - Blood and lymphatic system disorders 10051645 Idiopathic neutropenia PT
    14.1 10010331 - Congenital, familial and genetic disorders 10069819 Congenital neutropenia LLT
    14.1 10005329 - Blood and lymphatic system disorders 10053176 Cyclic neutropenia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2024-000442-10 Sponsor Protocol Number: A8081056 Start Date*: 2024-10-11
    Sponsor Name:Pfizer, Inc
    Full Title: CRIZOTINIB (XALKORI(REGISTERED)) EXPANDED ACCESS PROTOCOL FOR THE TREATMENT OF ADULT OR PEDIATRIC PATIENTS WITH SOLID OR HEMATOLOGIC MALIGNANCIES THAT HARBOR A CRIZOTINIB-SENSITIVE MOLECULAR ALTERA...
    Medical condition: Solid tumors
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-001678-40 Sponsor Protocol Number: CACZ885N2301E2 Start Date*: 2017-05-15
    Sponsor Name:Novartis pharma AG
    Full Title: An extension study of safety of canakinumab in Japanese patients with periodic fever syndromes
    Medical condition: Three disease of periodic fever syndromes, TNF receptor Associated Periodic Syndrome (TRAPS), Hyper IgDSyndrome (HIDS)/ Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2018-000368-27 Sponsor Protocol Number: MAN-ALG-18-001 Start Date*: 2018-10-22
    Sponsor Name:FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
    Full Title: ND
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-002856-33 Sponsor Protocol Number: ALN-TTRSC-002 Start Date*: 2013-11-15
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients with Transthyretin (TTR) Cardiac Amyloidosis
    Medical condition: Transthyretin (TTR) Cardiac Amyloidosis
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-000672-28 Sponsor Protocol Number: VX10-770-107 Start Date*: 2017-03-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Single-Blind, Placebo-Controlled Study to Evaluate the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis, the G551D Mutation, and FE...
    Medical condition: cystic fibrosis
    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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-001004-33 Sponsor Protocol Number: VX15-809-115 Start Date*: 2018-01-10
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for t...
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2007-006301-24 Sponsor Protocol Number: 06/143 Start Date*: 2008-02-21
    Sponsor Name:University College London, Joint Research Office
    Full Title: MULTI CENTRE CONTROLLED STUDY ON THE IMPACT OF STEM CELL DONATION EITHER AFTER MOBILISATION WITH GRANULOCYTE COLONY STIMULATING FACTOR OR BONE MARROW HARVEST ON UNRELATED BONE MARROW DONORS
    Medical condition: Study on the impact of stem cell donation either after mobilisation with granulocyte colony stimulating factor (GCSF) or bone marrow harvest on unrelated donors.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10042613 - Surgical and medical procedures 10051716 Peripheral blood stem cell apheresis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-000763-41 Sponsor Protocol Number: FBB-FAD-2014 Start Date*: 2014-08-20
    Sponsor Name:FUNDACIÓ CLÍNIC PER A LA RECERCA BIOMÈDICA
    Full Title: Study of genetic Alzheimer?s disease mutation carriers in preclinical stages of the disease: 18F-Florbetaben Positron Emission Tomography study.
    Medical condition: Healthy voulnteers carrying a mutation in at least one of the following genes: PSEN1, PSEN2 or APP.
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004852 10001897 Alzheimer's disease (incl subtypes) HLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-001229-34 Sponsor Protocol Number: ALN-TTRSC-003 Start Date*: 2014-08-01
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients with Transthyretin (TTR) Cardiac Amyloidosis Who Have Previo...
    Medical condition: Transthyretin (TTR) Cardiac Amyloidosis
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-001309-34 Sponsor Protocol Number: VX14-809-106 Start Date*: 2017-03-29
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR ...
    Medical condition: Cystic Fibrosis
    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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000673-37 Sponsor Protocol Number: VX12-770-113 Start Date*: 2017-03-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted
    Medical condition: cystic fibrosis
    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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-004996-33 Sponsor Protocol Number: VX15-809-114 Start Date*: 2017-05-03
    Sponsor Name:Vertex Pharmaceuticals Incoporated
    Full Title: A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallelgroup, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del-CFTR Mutation
    Medical condition: Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation
    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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    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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