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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 2 of 71.
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    EudraCT Number: 2017-000266-29 Sponsor Protocol Number: AVXS-101-CL-302 Start Date*: 2018-06-12
    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: Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) SE (Prematurely Ended) BE (Completed) ES (Prematurely Ended) NL (Prematurely Ended) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-004761-33 Sponsor Protocol Number: CRO1881 Start Date*: 2012-03-08
    Sponsor Name:Imperial College
    Full Title: A randomised double-blind placebo-controlled Phase 2B clinical trial of repeated application of gene therapy in patients with cystic fibrosis
    Medical condition: Cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011763 Cystic fibrosis lung PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-003965-11 Sponsor Protocol Number: CC-TT-IMA-14 Start Date*: 2015-11-18
    Sponsor Name:University Medical Center Utrecht
    Full Title: Targeted therapy with Imatinib for treatment of poor prognosis mesenchymal-type resectable colon cancer: a proof-of-concept study in the preoperative window period.
    Medical condition: colon cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-000677-23 Sponsor Protocol Number: CLCL161A2201 Start Date*: 2012-10-24
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A Phase II, multi-center, open-label, neoadjuvant, randomized study of weekly paclitaxel with or without LCL161 in patients with triple negative breast cancer
    Medical condition: Triple negative breast cancer
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (Completed) ES (Completed) IE (Completed) IT (Completed) BE (Completed) CZ (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2005-001834-34 Sponsor Protocol Number: 4919 Start Date*: 2006-03-14
    Sponsor Name:Oxford Radcliffe Hospitals NHS Trust
    Full Title: Phase 2 trial of neo-adjuvant Oxaliplatin and 5-fluorouracil in oesophageal cancer
    Medical condition: Operable oesophageal cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-001366-14 Sponsor Protocol Number: 207757 Start Date*: 2017-09-14
    Sponsor Name:GLAXOSMITHKLINE RESEARCH AND DEVELOPMENT
    Full Title: A long-term safety and efficacy follow-on study in participants with transfusion dependent β-thalassemia who have previously received GSK2696277 (autologous hematopoietic stem cells genetically mod...
    Medical condition: Beta-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2021-002873-24 Sponsor Protocol Number: RM-493-035 Start Date*: 2022-05-16
    Sponsor Name:Rhythm Pharmaceuticals Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melano...
    Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing) DE (Trial now transitioned) GR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-003967-21 Sponsor Protocol Number: MGT009 Start Date*: 2017-06-09
    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 (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigme...
    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
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-006224-21 Sponsor Protocol Number: IJB-BCTL-20119167 Start Date*: 2013-06-20
    Sponsor Name:Jules Bordet Institute
    Full Title: A pre-operative window study evaluating Denosumab, a RANK-Ligand (RANKL) inhibitor and its biological effects in young pre-menopausal women diagnosed with early breast cancer
    Medical condition: We have designed this study to investigate if denosumab can modulate a number of biological processes including prolferation, RANK signaling and the mammary stem cell subpopulation.
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004864 10006190 Breast cancer invasive NOS LLT
    Population Age: Adults Gender: Female
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-001730-26 Sponsor Protocol Number: 205756 Start Date*: 2017-10-30
    Sponsor Name:Orchard Therapeutics (Europe) Ltd
    Full Title: A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic...
    Medical condition: Metachromatic Leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy 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: 2009-011152-22 Sponsor Protocol Number: GTG003.08 Start Date*: Information not available in EudraCT
    Sponsor Name:GENETHON
    Full Title: Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome
    Medical condition: Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autolo...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2020-001235-27 Sponsor Protocol Number: AVXS-101-CL-101 Start Date*: 2020-04-06
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101
    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: 2014-002222-12 Sponsor Protocol Number: G1XCGD.02 Start Date*: 2015-10-08
    Sponsor Name:Genethon
    Full Title: A phase I/II, non-randomized, monocentric, open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-Linked Chronic Granulomatous Disease.
    Medical condition: Chronic Granulomatous Disease (CGD) is a rare inherited disorder (1/250,000) of the phagocytes characterized by the inability of phagocytes (monocytes and neutrophils) to produce reactive oxygen sp...
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2005-001698-89 Sponsor Protocol Number: Monet Start Date*: 2006-06-08
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust
    Full Title: Molecular profiling of postmenopausal women with breast cancer on neoadjuvant exemestane or tamoxifen
    Medical condition: Early or locally advanced breast cancer
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-004024-11 Sponsor Protocol Number: SHP648-101 Start Date*: 2020-05-04
    Sponsor Name:Baxalta Innovations GmbH
    Full Title: An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects
    Medical condition: Hemophilia B is a X-linked recessive bleeding disorder caused by mutations in the gene encoding clotting factor IX (FIX) that result in disruption of the normal clotting pathway. Hemophilia B affec...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: DE (Prematurely Ended) HU (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-004860-39 Sponsor Protocol Number: TIGET-BTHAL Start Date*: 2015-03-04
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment ...
    Medical condition: Beta talassemia trasfusione dipendente
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-001528-32 Sponsor Protocol Number: ARCO-Homestudy Start Date*: 2020-05-14
    Sponsor Name:ISTITUTO NAZIONALE PER LE MALATTIE INFETTIVE "LAZZARO SPALLANZANI"
    Full Title: Adaptive Randomized trial for therapy of COrona virus disease 2019 at home with oral antivirals
    Medical condition: Coronavirus Covid 19 infection
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004855 10047468 Viral lower respiratory tract infections HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-003856-59 Sponsor Protocol Number: MGT007 Start Date*: 2017-04-12
    Sponsor Name:MeiraGTX UK II Ltd
    Full Title: Long-term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene thera...
    Medical condition: Achromatopsia caused by mutations in the CNGB3 gene
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001825-28 Sponsor Protocol Number: R119513 Start Date*: 2019-09-24
    Sponsor Name: The University of Manchester
    Full Title: Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscul...
    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: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-000898-20 Sponsor Protocol Number: MGT004 Start Date*: 2016-10-04
    Sponsor Name:MeiraGTx UK Ltd
    Full Title: Long term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/5-OPTIRPE65) for gene therapy of adults a...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    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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