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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 7 of 71.
    «« First « Previous 3  4  5  6  7  8  9  10  11  Next» Last»»
    EudraCT Number: 2014-001924-31 Sponsor Protocol Number: STH18166 Start Date*: 2014-08-26
    Sponsor Name:Sheffield Teaching Hospitals NHS Foundation Trust
    Full Title: Exploring novel mechanisms of vaccine failure and induction of pulmonary immunity following live attenuated influenza vaccination in HIV infected and uninfected individuals: a pilot study.
    Medical condition: HIV (effect of HIV on the response to live attenuated intranasal influenza vaccine)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004862 10020160 HIV disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2007-001800-20 Sponsor Protocol Number: PADBelch07 Start Date*: 2007-08-10
    Sponsor Name:University of Dundee
    Full Title: A randomised, placebo-controlled trial of the effect of pioglitazone on vascular endothelial function and novel biomarkers for macrovascular events in diabetics with established peripheral arterial...
    Medical condition: The study involves patients with type 2 Diabetes and Peripheral Arterial Disease.The overall objective of the research proposal is to determine the occurrence of biomarkers that typify the metaboli...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2010-019873-13 Sponsor Protocol Number: 1200/002 Start Date*: 2010-06-07
    Sponsor Name:UCL Cliniques Universitaires Saint Luc
    Full Title: Comparative study of the clinical response and cardiorespiratory endurance in early rheumatoid arthritis patients treated with Tociluzimab or Methotrexate Addendum protocol : Global gene expression...
    Medical condition: early rheumatoid arthritis
    Disease: Version SOC Term Classification Code Term Level
    12.1 10039075 Rheumatoid arthritis and associated conditions HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2006-001707-11 Sponsor Protocol Number: M06HER Start Date*: 2007-06-07
    Sponsor Name:NKI-AvL
    Full Title: Prospective, randomized, pharmacological intervention study; evaluating the effect of the angiotensin II-receptor (AT1) blocker candesartan versus placebo in prevention of trastuzumab-associated ca...
    Medical condition: WHO: 0-2 Serum creatinine <140 umol/l Thyroid stimulating hormone between 0.5-3.9 MU/l. Blood pressure systolic ≥ 140 mmHg diastolic ≥ 90 mmHg is acceptable at randomization. LVEF  50% assessed b...
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-004869-14 Sponsor Protocol Number: CLL19H1 Start Date*: 2019-04-15
    Sponsor Name:Dept. of hematology, Herlev Hospital
    Full Title: Peptide vaccination with PD-L1 and PD-L2 peptides in untreated chronic lymphatic leukemia.
    Medical condition: Chronic lymphocytic leukemia with un-mutated IGHV
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008958 Chronic lymphocytic leukaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2018-002267-25 Sponsor Protocol Number: SEOTP-2018 Start Date*: 2018-07-18
    Sponsor Name:Fakultní nemocnice Brno
    Full Title: SAFETY AND EFFICACY OF OLANZAPINE TREATMENT IN PSYCHOSIS: EFFECT OF GENETIC AND EPIGENETIC FACTORS – COVARIATES OF TREATMENT RESPONSE
    Medical condition: Psychoses
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: CZ (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-000980-21 Sponsor Protocol Number: PTC124-GD-048-DMD Start Date*: 2021-06-24
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy
    Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-003226-16 Sponsor Protocol Number: BP-404 Start Date*: 2017-02-03
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-002204-27 Sponsor Protocol Number: GS030_CLIN_001 Start Date*: 2017-12-22
    Sponsor Name:GENSIGHT-BIOLOGICS
    Full Title: A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa
    Medical condition: Retinitis Pigmentosa
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-003705-34 Sponsor Protocol Number: RDC-PDE6A-01 Start Date*: 2019-07-02
    Sponsor Name:Universitätsklinikum Tübingen
    Full Title: PIGMENT – PDE6A gene therapy for retinitis pigmentosa
    Medical condition: PDE6A-linked retinitis pigmentosa
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-001461-36 Sponsor Protocol Number: CT-AMT-130-02 Start Date*: 2021-06-30
    Sponsor Name:uniQure biopharma B.V.
    Full Title: A Phase Ib/II Randomised, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Ther...
    Medical condition: Huntington Disease
    Disease: Version SOC Term Classification Code Term Level
    27.1 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: PL (Trial now transitioned) DE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2005-000106-31 Sponsor Protocol Number: 2958 Start Date*: 2005-03-15
    Sponsor Name:Royal Liverpool University Hospital Clinical Trials 4th floor Linda McCartney Centre
    Full Title: IMPACT OF CYP2D6, SEROTONIN TRANSPORTER, 5HT1, 5HT2 AND 5HT3 RECEPTOR AND MU RECEPTOR GENE POLYMORPHISMS ON EFFICACY AND TOXICITY OF TRAMADOL
    Medical condition: OSTEOPOROSIS
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-002636-82 Sponsor Protocol Number: OTL-200-07 Start Date*: 2019-12-02
    Sponsor Name:Orchard Therapeutics (Europe) Limited
    Full Title: An open label, non-randomised trial to evaluate the safety and efficacy of a single infusion of OTL-200 in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD)
    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: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-003299-10 Sponsor Protocol Number: M13DAP Start Date*: 2014-01-14
    Sponsor Name:The Netherlands Cancer Institute
    Full Title: Phase I/II study with the combination of dacomitinib and PD-0325901 in metastatic KRAS mutation positive non-small cell lung cancer
    Medical condition: non-small cell lung cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-001648-79 Sponsor Protocol Number: KFE13.10 Start Date*: 2013-07-22
    Sponsor Name:Odense University Hospital
    Full Title: Epirubicin as 2nd line treatment to patients with TOP2A gene amplified and oxaliplatin refractory metastatic colerectal cancer
    Medical condition: TOP2A gene amplified and oxaliplatin refractory metastatic colorectal cancer
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061451 Colorectal cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2015-004592-74 Sponsor Protocol Number: HOLOGENE7(HTA-HG7-01) Start Date*: 2016-06-01
    Sponsor Name:Holostem Terapie Avanzate s.r.l.
    Full Title: PROSPECTIVE, OPEN LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETR...
    Medical condition: RDEB is characterized by generalized skin blistering, erosions, crusts, atrophic scarring, onychodystrophy and loss of nails, mutilating pseudosyndactyly of hands and feet, as well as oral cavity l...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-000095-17 Sponsor Protocol Number: HOLOGENE17 Start Date*: 2017-08-17
    Sponsor Name:Holostem Terapie Avanzate s.r.l.
    Full Title: PROSPECTIVE, OPEN-LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETR...
    Medical condition: JEB is genetically and clinically heterogeneous characterized by mutations in COL17A1,encoding for Collagen17 protein. COLXVII is a collagenous transmembrane type II protein component of the hemide...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-002790-35 Sponsor Protocol Number: C12-48 Start Date*: 2019-06-04
    Sponsor Name:INSERM
    Full Title: Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector
    Medical condition: The trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004858 10074980 Epidermolysis bullosa aquisita LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: View results
    EudraCT Number: 2018-003584-53 Sponsor Protocol Number: Debio1347-201 Start Date*: 2019-02-05
    Sponsor Name:Debiopharm International SA
    Full Title: A Phase II basket study of the oral selective pan-FGFR inhibitor Debio 1347 in subjects with solid tumors harboring a fusion of FGFR1, FGFR2 or FGFR3
    Medical condition: Three cohorts will be included consisting of subjects with biliary tract cancer (Cohort 1), urothelial cancer (Cohort 2) and all other solid tumor histologies not included in Cohorts 1-2 such as NS...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) GR (Completed) AT (Completed) NL (Ongoing) GB (GB - no longer in EU/EEA) CZ (Prematurely Ended) DK (Completed) NO (Completed) BG (Completed) FI (Completed) ES (Temporarily Halted) HR (Completed) RO (Completed)
    Trial results: View results
    EudraCT Number: 2017-004246-20 Sponsor Protocol Number: 20810 Start Date*: 2018-03-06
    Sponsor Name:Bayer Consumer Care AG
    Full Title: A Phase 1 Study of the TRK Inhibitor Selitrectinib (BAY 2731954) in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers Formerly: A Phase 1/2 Study of the TRK Inhibitor LOXO-1...
    Medical condition: NTRK fusion cancers previously treated with a TRK inhibitor
    Disease: Version SOC Term Classification Code Term Level
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10049516 Malignant tumor LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10007958 Central nervous system neoplasm PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed) FR (Completed) DE (Completed) ES (Completed) GB (GB - no longer in EU/EEA) IE (Completed) IT (Prematurely Ended) BE (Completed)
    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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