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Clinical trials for gene 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   44393   clinical trials with a EudraCT protocol, of which   7405   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 ).  
     
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    1,407 result(s) found for: gene therapy. Displaying page 4 of 71.
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    EudraCT Number: 2007-000684-16 Sponsor Protocol Number: 03MI10 Start Date*: 2010-01-21
    Sponsor Name:Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health
    Full Title: Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector.
    Medical condition: X-Linked severe combined Immunodeficiency (SCID-X1)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010099 Combined immunodeficiency LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-004272-29 Sponsor Protocol Number: 14-MI-10 Start Date*: 2015-03-30
    Sponsor Name:Great Ormond Street Hospital NHS foundation Trust
    Full Title: Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent ...
    Medical condition: Fanconi Anaemia
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2004-005054-31 Sponsor Protocol Number: 24531 Start Date*: 2005-07-15
    Sponsor Name:Serono International S.A.
    Full Title: A phase IV open-label study of predictive markers in Growth Hormone Deficient and Turner Syndrome prepubertal children treated with Saizen
    Medical condition: - growth failure in children caused by decreased or absent secretion of endogenous growth hormone. - growth failure in girls with gonadal dysgenesis (Turner Syndrome), confirmed by chromosomal anal...
    Disease: Version SOC Term Classification Code Term Level
    7.1 10056438 PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) FI (Completed) ES (Completed) DE (Completed) AT (Completed) SE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-002680-26 Sponsor Protocol Number: RP-L201-0218 Start Date*: 2018-11-27
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lenti...
    Medical condition: Leukocyte Adhesion Deficiency-I (LAD-I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018137 Genetic anomalies of leukocytes LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-002430-23 Sponsor Protocol Number: TigetT10_MPS1H Start Date*: 2018-03-14
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene ...
    Medical condition: Mucopolysaccharidosis type I Hurler
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2005-000459-14 Sponsor Protocol Number: VUB/MedOnc/05-001 Start Date*: 2007-07-02
    Sponsor Name:Oncologisch Centrum, Academisch Ziekenhuis Vrije Universiteit Brussel
    Full Title: A Stratified Phase II Study of Cetuximab (ErbituxTM) for the Treatment of Recurrent Glioblastoma Multiforme / Estudio de fase II estratificado de cetuximab para el tratamiento del glioblastoma mult...
    Medical condition: patients with a recurrent glioblastoma multiforme following preceding surgery, radiation therapy and chemotherapy
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-003072-39 Sponsor Protocol Number: NTUH-AADC-011 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-004810-16 Sponsor Protocol Number: TPU-TAS-120-101 Start Date*: 2014-04-10
    Sponsor Name:Taiho Oncology Inc
    Full Title: PHASE 1/2 STUDY OF TAS-120 IN PATIENTS WITH ADVANCED SOLID TUMORS HARBORING FGF/FGFR ABERRATIONS
    Medical condition: Advanced solid tumors
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10065147 Malignant solid tumor LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Completed) DE (Completed) NL (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2009-012595-27 Sponsor Protocol Number: A8081007 Start Date*: 2010-01-20
    Sponsor Name:Pfizer Inc. 235 East 42nd Street, New York, NY 10017
    Full Title: PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF THE EFFICACY AND SAFETY OF PF 02341066 VERSUS STANDARD OF CARE CHEMOTHERAPY (PEMETREXED OR DOCETAXEL) IN PATIENTS WITH ADVANCED NON-SMALL CELL LUNG CANCER (...
    Medical condition: ADVANCED NON-SMALL CELL LUNG CANCER (NSCLC) HARBORING A TRANSLOCATION OR INVERSION EVENT INVOLVING THE ANAPLASTIC LYMPHOMA KINASE (ALK) GENE LOCUS
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) DE (Completed) PL (Completed) ES (Completed) HU (Completed) FR (Completed) GR (Completed) IT (Completed) IE (Completed) BG (Completed) SE (Completed)
    Trial results: View results
    EudraCT Number: 2013-005086-40 Sponsor Protocol Number: J1214 Start Date*: 2014-05-21
    Sponsor Name:Sidney kimmel Comprehensive Cancer Center at Johns Hopkins
    Full Title: A multi-institutional open label, trial evaluating the efficacy of Gemcitabine and Docetaxel in patients with relapsed or refractory metastatic colorectal adenocarcinoma with methylated CHFR and/or...
    Medical condition: relapsed or refractory metastatic colorectal adenocarcinoma with methylation of CHFR and/or microsatellite instability
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10010030 Colorectal cancer recurrent PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-000517-33 Sponsor Protocol Number: RP-L201-0318 Start Date*: 2020-07-14
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Len...
    Medical condition: Leukocyte Adhesion Deficiency-I (LAD-I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018137 Genetic anomalies of leukocytes LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2020-003678-28 Sponsor Protocol Number: AVXS-101-CL-102 Start Date*: 2020-09-15
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non- Ambulatory Patients with Spinal Muscular Atrophy
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-001019-22 Sponsor Protocol Number: KAT-PAD101 Start Date*: 2012-10-26
    Sponsor Name:A.I.Virtanen Insitute, University of Kuopio
    Full Title: Treatment of chronic critical lower limb ischemia with combination of surgical bypass operation and adenoviral VEGF-DdNdC intramuscular gene transfer A Randomized Single-Blinded Placebo-control...
    Medical condition: Critical Chronic lower limb ischemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10047065 - Vascular disorders 10058069 Critical limb ischemia LLT
    Population Age: Elderly Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-002502-31 Sponsor Protocol Number: RP-L102-0118 Start Date*: 2018-10-31
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemi...
    Medical condition: Fanconi anemia (subtype A)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2012-000394-23 Sponsor Protocol Number: SOLTI-1007 Start Date*: 2012-08-06
    Sponsor Name:SOLTI
    Full Title: A Phase II, open-label, single-arm, exploratory pharmacogenomic study of single agent eribulin (HALAVEN®) as neoadjuvant treatment for operable Stage I-II HER2 non-overexpressing breast cancer
    Medical condition: Patients at Stage I-II HER2-negative breast cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) DE (Completed) PT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-004189-33 Sponsor Protocol Number: REK322442 Start Date*: 2013-11-28
    Sponsor Name:Haukeland University Hospital
    Full Title: ULTRADIAN SUBCUTANEOUS HYDROCORTISONE (USHI) INFUSION IN ADDISON’S DISEASE
    Medical condition: Addison`s disease
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NO (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-005525-39 Sponsor Protocol Number: CCR4449 Start Date*: 2016-05-19
    Sponsor Name:The Royal Marsden NHS Foundation Trust
    Full Title: Proof-of-concept study of ibrutinib in c-MYC and HER2 amplified gastrooesophageal carcinoma
    Medical condition: Her2 or cMYC positive advanced oesophagogastric carcinomas
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-000156-18 Sponsor Protocol Number: 301OTC02 Start Date*: 2018-08-15
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Lat...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-003111-10 Sponsor Protocol Number: D-00272-CT2014002 Start Date*: 2016-06-01
    Sponsor Name:Cell Medica Ltd
    Full Title: A single arm Phase I/II study of the safety and efficacy of gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML) who have failed to achieve...
    Medical condition: Acute myeloid leukaemia and myelodysplastic syndromes in adults
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000880 Acute myeloid leukaemia PT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028533 Myelodysplastic syndrome PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) BE (Completed)
    Trial results: View results
    EudraCT Number: 2021-001811-94 Sponsor Protocol Number: M18-868 Start Date*: 2022-01-28
    Sponsor Name:AbbVie Deutschland GmbH & Co. KG
    Full Title: A Phase 3 Open-Label, Randomized, Controlled, Global Study of Telisotuzumab Vedotin (ABBV-399) Versus Docetaxel in Subjects with Previously Treated c-Met Overexpressing, EGFR Wildtype, Locally Adva...
    Medical condition: C-Met overexpressing EGFR wildtype, non-squamous non-small cell lung cancer
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10079440 Non-squamous non-small cell lung cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SK (Trial now transitioned) CZ (Trial now transitioned) SE (Trial now transitioned) BE (Trial now transitioned) HU (Completed) FR (Trial now transitioned) RO (Trial now transitioned) AT (Trial now transitioned) IT (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing) PT (Trial now transitioned) DK (Trial now transitioned)
    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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