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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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    1,407 result(s) found for: gene therapy. Displaying page 3 of 71.
    « Previous 1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2021-000265-33 Sponsor Protocol Number: 3000LM Start Date*: 2021-02-25
    Sponsor Name:The Rotterdam Eye Hospital
    Full Title: Prospective study on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna®) in patients with RPE65-associated inherited retinal degenerations
    Medical condition: RPE65-associated inherited retinal degeneration
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-001465-24 Sponsor Protocol Number: GC-LTFU-001 Start Date*: 2018-02-26
    Sponsor Name:Celgene Corporation
    Full Title: Long-Term Follow-up Protocol for Subjects Treated with Gene-Modified T cells.
    Medical condition: Defined by parent protocol. The study will enroll all adult and paediatric subjects who received at least one genetically modified T cells infusion in a previous Celgene sponsored study.
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10025631 Malignant lymphoid neoplasm NOS LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) DE (Trial now transitioned) ES (Ongoing) NL (Trial now transitioned) AT (Trial now transitioned) FR (Trial now transitioned) FI (Trial now transitioned) SE (Trial now transitioned) GB (GB - no longer in EU/EEA) NO (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004266-18 Sponsor Protocol Number: LTF-307 Start Date*: 2020-09-03
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
    Medical condition: Subjects with sickle-cell disease treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety an...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10040648 Sickle cell SC disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-001656-19 Sponsor Protocol Number: RP-L301-0119 Start Date*: 2019-09-19
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon O...
    Medical condition: Pyruvate kinase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-004448-12 Sponsor Protocol Number: ARQ087-301 Start Date*: 2017-12-18
    Sponsor Name:ARQULE INC
    Full Title: A Pivotal Trial of ARQ 087 in Subjects with FGFR2 Gene Fusion Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
    Medical condition: Inoperable or advanced FGFR2 gene fusion positive intrahepatic cholangiocarcinoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10073078 Intrahepatic cholangiocarcinoma recurrent LLT
    20.0 100000004864 10073077 Intrahepatic cholangiocarcinoma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) DE (Completed) IE (Completed) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2006-001197-24 Sponsor Protocol Number: D791AC00011 Start Date*: 2006-09-18
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase II, Double-blind, Randomised, Parallel Group, Multi-centre Study Comparing gefitinib 250 mg (IRESSA™) with erlotinib 150 mg (Tarceva®) in Previously Treated Patients with Locally Advanced o...
    Medical condition: Non-small Cell Lung Cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) AT (Completed) DE (Completed) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-000095-28 Sponsor Protocol Number: H3E-MC-JMHW Start Date*: 2017-03-02
    Sponsor Name:Eli Lilly and Company
    Full Title: A Phase II Study of Pemetrexed in Children with Recurrent Malignancies
    Medical condition: Recurrent Malignancies (recurrent solid tumors): Target tumor types were osteosarcoma, Ewing sarcoma/peripheral PNET, rhabdomyosarcoma, neuroblastoma (measurable disease), neuroblastoma (metaiodob...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003138-17 Sponsor Protocol Number: P1200_51 Start Date*: 2020-01-17
    Sponsor Name:Cliniques universitaires Saint-Luc
    Full Title: A 24 weeks proof of concept study to evaluate the clinical, MRI and synovial tissue benefit of Sarilumab in active RA patients despite TNF agents
    Medical condition: Medical condition – Rheumatoid Arthritis
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004859 10003268 Arthritis rheumatoid LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002255-37 Sponsor Protocol Number: MGT-RPGR-022 Start Date*: 2022-05-26
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IE (Prematurely Ended) NL (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) DK (Trial now transitioned) DE (Prematurely Ended) IT (Trial now transitioned) Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2015-001443-36 Sponsor Protocol Number: ARQ087-101 Start Date*: 2015-08-05
    Sponsor Name:ARQULE INC
    Full Title: A Phase 1/2 Study of ARQ 087 in Adult Subjects with Advanced Solid Tumors with FGFR Genetic Alterations, Including Intrahepatic Cholangiocarcinoma with FGFR2 Gene Fusion
    Medical condition: Advanced Solid Tumors with FGFR Genetic Alterations, Including Intrahepatic Cholangiocarcinoma with FGFR2 Gene Fusion
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004864 10065143 Malignant solid tumour LLT
    20.0 100000004864 10073077 Intrahepatic cholangiocarcinoma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-002941-36 Sponsor Protocol Number: M11TCR Start Date*: 2012-04-02
    Sponsor Name:NKI-AVL
    Full Title: Multicenter phase 1/2a study using T-cell receptor gene therapy in metastatic melanoma
    Medical condition: Melanoma
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10025671 Malignant melanoma stage IV PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2007-003938-41 Sponsor Protocol Number: IMP 28364 Start Date*: 2007-09-25
    Sponsor Name:Merck Lipha Santé SAS
    Full Title: Open label pilot study on gene expression profiling of the endometrial tissue in patients undergoing Assisted Reproductive Technology (ART: only IVF) with GONAL-f.
    Medical condition: This study will be conducted in infertile women justifying ART (Assisted Reproductive Technology) treatment (only IVF - In Vitro Fecondation)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056204 In vitro fertilisation LLT
    Population Age: Adults Gender: Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2020-002873-88 Sponsor Protocol Number: MGT-RPGR-021 Start Date*: 2022-07-19
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IE (Completed) NL (Ongoing) BE (Completed) ES (Ongoing) FR (Completed) DK (Completed) DE (Prematurely Ended) IT (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-001654-65 Sponsor Protocol Number: PDY16967 Start Date*: 2021-11-30
    Sponsor Name:Sanofi-Aventis Recherche & Developpement
    Full Title: A Phase 2a, open label, two-part study to evaluate the mechanism of action of itepekimab (anti-IL-33 mAb) on airway inflammation in patients with chronic obstructive pulmonary disease (COPD)
    Medical condition: Chronic Obstructive Pulmonary Disease
    Disease: Version SOC Term Classification Code Term Level
    26.1 10038738 - Respiratory, thoracic and mediastinal disorders 10009033 Chronic obstructive pulmonary disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned)
    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: 2012-005036-28 Sponsor Protocol Number: 2012/41 Start Date*: 2013-04-12
    Sponsor Name:CENTRE ANTOINE LACASSAGNE
    Full Title: Pharmacogenetic study in castration-resistant prostate cancer patients treated with abiraterone acetate
    Medical condition: Prostate cancer metastatic
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036909 Prostate cancer metastatic PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2016-002364-13 Sponsor Protocol Number: M16VIB Start Date*: 2018-01-23
    Sponsor Name:Netherlands Cancer Institute- Antoni van Leeuwenhoek hospital (NKI-AVL)
    Full Title: MoTriColor: A phase II study of vinorelbine in advanced BRAF-like colon cancer
    Medical condition: colon carcinoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10052358 Colorectal cancer metastatic PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) BE (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-001725-26 Sponsor Protocol Number: X-CGD-Version2 Start Date*: 2013-03-12
    Sponsor Name:Johann Wolfgang Goethe-University
    Full Title: A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector
    Medical condition: Chronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10008906 Chronic granulomatous disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2013-002245-11 Sponsor Protocol Number: LTF-303 Start Date*: 2013-11-12
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
    Medical condition: Subjects treated with gene therapy drug products in a bluebird bio-sponsored or associated clinical study will be invited to participate in this long-term follow-up study to monitor the safety and ...
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2008-006649-18 Sponsor Protocol Number: 08/0214 Start Date*: 2011-11-16
    Sponsor Name:University College London
    Full Title: CMV TCR Gene Therapy: A Phase I Safety, Toxicity and Feasibility Study of Adoptive Immunotherapy with CMV TCR-transduced Donor-derived T cells for Recipients of Allogeneic Haematopoietic Stem Cell ...
    Medical condition: CMV reactivation/infection in post allogeneic haematopoietic stem cell transplant recipients. Allo-HSCT being performed for underlying haematological malignancy (eg, AML, ALL, NHL, Hodgkin Lymphoma...
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004865 10067859 Allogenic stem cell transplantation LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (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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