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Clinical trials for gene AND therapy

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   43859   clinical trials with a EudraCT protocol, of which   7284   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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    1,389 result(s) found for: gene AND therapy. Displaying page 1 of 70.
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    EudraCT Number: 2014-000274-20 Sponsor Protocol Number: GNT-WAS-03 Start Date*: 2014-05-14
    Sponsor Name:Genethon
    Full Title: LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08)
    Medical condition: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopeni...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10047992 Wiskott-Aldrich syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2015-001383-18 Sponsor Protocol Number: Start Date*: 2015-11-30
    Sponsor Name:University of Oxford
    Full Title: An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)
    Medical condition: Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the...
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004853 10008791 Choroideremia LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-003555-11 Sponsor Protocol Number: PD20180302 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE-HOPÏTAUX DE PARIS
    Full Title: A Phase 1/2 Open Label non randomized Study, multicentric, single arm evaluating the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the ...
    Medical condition: immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis)
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-004308-11 Sponsor Protocol Number: GTG002.07 Start Date*: 2010-01-11
    Sponsor Name:Genethon
    Full Title: PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME
    Medical condition: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia,...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10047992 Wiskott-Aldrich syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-000155-41 Sponsor Protocol Number: 1 Start Date*: 2019-01-10
    Sponsor Name:Department of Medical and Clinical Genetics, University of Helsinki
    Full Title: The effect of vitamin C on the gene methylation load in patients with TET2 mutations
    Medical condition: germline TET-2 gene mutation
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-024253-36 Sponsor Protocol Number: 10MI29 Start Date*: 2012-08-03
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1αS-ADA lentiviral vector mediated gene modification of autologus CD34+ cel...
    Medical condition: Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protect...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000012248 10066372 ADA deficiency LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2021-000077-83 Sponsor Protocol Number: SRP-9001-101 Start Date*: 2022-11-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Systemic gene delivery Phase I/IIa clinical trial for Duchenne muscular dystrophy using rAAVrh74.MHCK7.micro-dystrophin (microDys-IV-001)
    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
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-000349-59 Sponsor Protocol Number: 0244190615-02 Start Date*: 2014-05-12
    Sponsor Name:department of experimental and clinical medicine "F. Magrassi"
    Full Title: METAL (METformin in Advanced Lung cancer) study: PHASE II STUDY OF METFORMIN PLUS ERLOTINIB IN SECOND LINE THERAPY OF STAGE IV NON SMALL CELL LUNG CANCER (NSCLC) PATIENTS
    Medical condition: Patients with metastatic non small cell lung cancer in second line therapy
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-002343-14 Sponsor Protocol Number: RAG1-2019-01 Start Date*: 2020-09-25
    Sponsor Name:Leiden University Medical Center
    Full Title: PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY
    Medical condition: Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing) ES (Ongoing) PL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-003431-29 Sponsor Protocol Number: MGT012 Start Date*: 2019-06-24
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/8-hG1.7p.coCNGA3) for gene therapy of children with achromatopsia owing to defects in CNGA3
    Medical condition: Achromatopsia caused by mutations in the CNGA3 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-002290-35 Sponsor Protocol Number: MGT006 Start Date*: 2016-12-20
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing t...
    Medical condition: Achromatopsia caused by mutations in the CNGB3 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-000673-68 Sponsor Protocol Number: 16IC17 Start Date*: 2018-10-09
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan
    Medical condition: Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10069566 Severe combined immunodeficiency syndrome LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-002113-19 Sponsor Protocol Number: 42756493CAN2002 Start Date*: 2019-10-31
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Phase 2 Study of Erdafitinib in Subjects with Advanced Solid Tumors and FGFR Gene Alterations
    Medical condition: Advanced solid tumors (other than Urothelial tumors), and FGFR gene alterations.
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10065143 Malignant solid tumour LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PL (Completed) FR (Completed) DE (Trial now transitioned) ES (Ongoing) BE (Completed) IT (Ongoing) Outside EU/EEA SE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2007-007276-41 Sponsor Protocol Number: Ram_GEP_1 Start Date*: 2008-05-13
    Sponsor Name:Medizinische Universität Wien, Universitätsklinik für Klinische Pharmakologie
    Full Title: Gene expression profiling in skeletal muscle of healthy subjects treated with ramipril
    Medical condition: healthy young males
    Disease:
    Population Age: Adults Gender: Male
    Trial protocol: AT (Completed)
    Trial results: View results
    EudraCT Number: 2012-001638-33 Sponsor Protocol Number: IKEM-CEM-01 Start Date*: 2012-10-10
    Sponsor Name:Institute for Clinical and Experimental Medicine
    Full Title: Cholesterol 7alpha-hydroxylase polymorphism as a predictor cholesterolemia responsiveness
    Medical condition: hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10020604 Hypercholesterolemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: CZ (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-000925-38 Sponsor Protocol Number: UCL13/0076 Start Date*: 2017-01-04
    Sponsor Name:University College London (UCL)
    Full Title: GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding Factor VIII-V3
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2011-003212-22 Sponsor Protocol Number: 10MI30 Start Date*: 2013-06-03
    Sponsor Name:GOSH/ICH Joint Research & Development Office
    Full Title: Phase I study of ex-vivo lentiviral gene therapy for the inherited skin disease Netherton Syndrome
    Medical condition: Netherton Syndrome (NS)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10062909 Netherton's syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-000095-38 Sponsor Protocol Number: AVXS-101-CL-303 Start Date*: 2020-03-18
    Sponsor Name:AveXis, Inc.
    Full Title: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous ...
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-002093-27 Sponsor Protocol Number: GNT-016-MDYF Start Date*: 2020-11-30
    Sponsor Name:Genethon
    Full Title: Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple b...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-002209-25 Sponsor Protocol Number: 67652000PCR3002 Start Date*: 2020-10-12
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Phase 3 Randomized, Placebo-controlled, Double-blind Study of Niraparib in Combination with Abiraterone Acetate and Prednisone Versus Abiraterone Acetate and Prednisone for the Treatment of Parti...
    Medical condition: Deleterious Germline or Somatic Homologous Recombination Repair (HRR) Gene-Mutated Metastatic Castration-Sensitive Prostate Cancer (mCSPC)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036909 Prostate cancer metastatic PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) SE (Trial now transitioned) FR (Trial now transitioned) CZ (Trial now transitioned) HU (Trial now transitioned) BE (Trial now transitioned) NL (Ongoing) DK (Trial now transitioned) PL (Trial now transitioned) DE (Ongoing) PT (Ongoing) BG (Ongoing) IT (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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