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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 5 of 71.
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    EudraCT Number: 2005-005711-17 Sponsor Protocol Number: AGT4HB Start Date*: 2009-09-25
    Sponsor Name:St. Jude Children’s Research Hospital
    Full Title: AN OPEN LABEL DOSE-ESCALATION STUDY OF A SELF COMPLEMENTARY ADENO-ASSOCIATED VIRAL VECTOR (scAAV2/8-LP1-hFIXco) FOR GENE TRANSFER IN SUBJECTS WITH HEMOPHILIA B
    Medical condition: Gene therapy for Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004865 10001140 Adeno-associated in vivo gene therapy LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10061992 Haemophilia PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2008-002447-16 Sponsor Protocol Number: 111482 Start Date*: 2008-11-10
    Sponsor Name:GlaxoSmithKline Biologicals
    Full Title: A double-blind, randomized, placebo-controlled Phase III study to assess the efficacy of recMAGE-A3 + AS15 ASCI as adjuvant therapy in patients with MAGE-A3 positive resected stage III melanoma
    Medical condition: Patients with histologically proven stage IIIB or IIIC cutaneous melanoma with macroscopic lymph node involvement suitable for surgical resection. Macroscopic lymph node involvement is defined as c...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10025670 Malignant melanoma stage III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Prematurely Ended) DE (Prematurely Ended) BE (Completed) CZ (Prematurely Ended) FR (Completed) NL (Prematurely Ended) IT (Prematurely Ended) SE (Prematurely Ended) EE (Prematurely Ended) ES (Completed) AT (Prematurely Ended) GR (Prematurely Ended) BG (Prematurely Ended) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-003616-13 Sponsor Protocol Number: TPX-0005-01 Start Date*: 2020-05-12
    Sponsor Name:Turning Point Therapeutics, Inc.
    Full Title: A Phase 1/2, Open-Label, Multi-Center, First-in-Human Study of the Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of TPX-0005 in Patients with Advanced Solid Tumors Harboring ALK, ...
    Medical condition: advanced solid tumors
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10065252 Solid tumor LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) GB (GB - no longer in EU/EEA) NL (Trial now transitioned) DE (Trial now transitioned) PL (Trial now transitioned) ES (Ongoing) BE (Trial now transitioned) HU (Trial now transitioned) DK (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000425-31 Sponsor Protocol Number: MGT010 Start Date*: 2018-08-30
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: Long term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy o...
    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: 2008-004457-15 Sponsor Protocol Number: EMD 121974-012 Start Date*: 2010-08-13
    Sponsor Name:Merck KGaA
    Full Title: Cilengitide in subjects with newly diagnosed glioblastoma multiforme and unmethylated MGMT gene promoter - a multicenter, open-label Phase II study, investigating two cilengitide regimens in combin...
    Medical condition: Newly diagnosed glioblastoma multiforme with unmethylated MGMT gene promoter status
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018337 Glioblastoma multiforme LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) IT (Prematurely Ended) FR (Completed) HU (Completed) AT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2010-022978-14 Sponsor Protocol Number: A8081013 Start Date*: 2011-02-10
    Sponsor Name:Pfizer Inc, 235 East 42nd Street, New York, NY 10017
    Full Title: PHASE 1B OPEN-LABEL STUDY OF THE SAFETY AND CLINICAL ACTIVITY OF CRIZOTINIB (PF-02341066) IN TUMORS WITH GENETIC EVENTS INVOLVING THE ANAPLASTIC LYMPHOMA KINASE (ALK ) GENE LOCUS
    Medical condition: Treatment of tumors with translocation, mutation, or amplification of the anaplastic lymphoma kinase (ALK)gene locus.
    Disease: Version SOC Term Classification Code Term Level
    13.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065252 Solid tumor LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    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
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-019577-16 Sponsor Protocol Number: EGF114299 Start Date*: 2010-12-21
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A Phase III trial to compare the safety and efficacy of lapatinib plus trastuzumab plus an aromatase inhibitor (AI) versus trastuzumab plus an AI versus lapatinib plus an AI as first- or second-lin...
    Medical condition: HER2-positive metastatic breast cancer who have received prior trastuzumab and endocrine therapies
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065430 HER-2 positive breast cancer PT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: DE (Prematurely Ended) HU (Completed) IE (Prematurely Ended) PL (Completed) BE (Completed) BG (Prematurely Ended) NO (Prematurely Ended) GB (Completed) LT (Prematurely Ended) PT (Prematurely Ended) ES (Ongoing) GR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-003430-25 Sponsor Protocol Number: 101HEMB02 Start Date*: 2017-05-18
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Sever...
    Medical condition: Moderate/severe to severe hemophilia B
    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: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2007-004344-78 Sponsor Protocol Number: EMD121974-011 Start Date*: 2008-08-28
    Sponsor Name:Merck KGaA
    Full Title: Cilengitide in subjects with newly diagnosed glioblastoma multiforme and methylated MGMT gene promoter – a multicenter, open-label, controlled Phase III study, testing cilengitide in combination wi...
    Medical condition: Newly diagnosed, histologically proven GBM with proven methylated MGMT gene promoter status
    Disease: Version SOC Term Classification Code Term Level
    15.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10018337 Glioblastoma multiforme PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) GB (Completed) AT (Completed) DE (Completed) CZ (Completed) FR (Completed) ES (Prematurely Ended) IT (Prematurely Ended) SK (Completed) HU (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2009-017346-32 Sponsor Protocol Number: 201228 Start Date*: 2010-03-15
    Sponsor Name:Fondazione Telethon
    Full Title: A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME
    Medical condition: Wiskott-Aldrich Syndrom
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021428 - Immune system disorders 10061598 Immunodeficiency PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-002991-42 Sponsor Protocol Number: HCV-13-01 Start Date*: 2013-12-19
    Sponsor Name:Foundation for Liver Research
    Full Title: Effect of ASV and DCV therapy on the quality of immune status in chronic HCV patients Investigator initiated research proposal nr. AI447-108
    Medical condition: Chronic hepatitis C
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005843-21 Sponsor Protocol Number: KT-US-982-5968 Start Date*: 2022-02-08
    Sponsor Name:KITE PHARMA INC.
    Full Title: Long-term Follow-up Study for Participants of Kite Sponsored Interventional Studies Treated With Gene-Modified Cells
    Medical condition: B-cell malignancies and Acute Myeloid Leukemia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10026801 Mantle cell lymphoma refractory PT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008977 Chronic lymphocytic leukemia recurrent LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008978 Chronic lymphocytic leukemia refractory LLT
    23.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065856 Non-Hodgkin's lymphoma unspecified histology indolent PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10012822 Diffuse large B-cell lymphoma refractory PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10060558 Acute myeloid leukemia recurrent LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10081514 Acute myeloid leukemia refractory LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10063621 Acute lymphoblastic leukaemia recurrent LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10006599 Burkitt's lymphoma refractory PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036714 Primary mediastinal large B-cell lymphoma refractory PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) FR (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005736-30 Sponsor Protocol Number: CDAII-LUSPA_FA02-2020 Start Date*: 2023-07-17
    Sponsor Name:FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    Full Title: A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll).
    Medical condition: Congenital Dyserythropoietic Anemia type II (CDAII)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-001266-26 Sponsor Protocol Number: GS-LHON-CLIN-03B Start Date*: 2016-01-21
    Sponsor Name:GENSIGHT-BIOLOGICS
    Full Title: A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for more than 6 months a...
    Medical condition: Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-003385-84 Sponsor Protocol Number: GO40782(RXDX-101-02) Start Date*: 2016-03-15
    Sponsor Name:F. Hoffman-La Roche Ltf
    Full Title: AN OPEN-LABEL, MULTICENTER, GLOBAL PHASE 2 BASKET STUDY OF ENTRECTINIB FOR THE TREATMENT OF PATIENTS WITH LOCALLY ADVANCED OR METASTATIC SOLID TUMORS THAT HARBOR NTRK1/2/3, ROS1, OR ALK GENE REARRA...
    Medical condition: Locally Advanced or Metastatic Solid Tumors that Harbor NTRK1/2/3, ROS1, or ALK Gene Rearrangements
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10065147 Malignant solid tumor LLT
    21.1 100000004864 10065252 Solid tumor LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) DE (Trial now transitioned) ES (Ongoing) NL (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-002209-39 Sponsor Protocol Number: M14LTK Start Date*: 2014-07-30
    Sponsor Name:The Netherlands Cancer Institute
    Full Title: Phase I/II study with lapatinib plus trametinib in patients with metastatic KRAS mutant non-small cell lungcancer
    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: 2009-017349-77 Sponsor Protocol Number: TIGET-MLD Start Date*: 2010-03-15
    Sponsor Name:FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
    Full Title: A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy
    Medical condition: metachromatic leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 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: 2017-000789-31 Sponsor Protocol Number: RGH201 Start Date*: 2018-02-09
    Sponsor Name:Kuopio University Hospital Heart Center
    Full Title: Clinical development and proof of principle testing of new regenerative VEGF-D therapy for cost-effective treatment of refractory angina. A phase II randomized, double-blinded, placebo-controlled s...
    Medical condition: Refractory angina
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004849 10064262 Refractory angina pectoris LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned) FI (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-002095-40 Sponsor Protocol Number: B2151005 Start Date*: 2014-06-12
    Sponsor Name:Pfizer Inc.,235 East 42nd Street, New York, NY 10017
    Full Title: A RANDOMIZED PHASE 2 STUDY OF PF-05212384 PLUS IRINOTECAN VERSUS CETUXIMAB PLUS IRINOTECAN IN PATIENTS WITH KRAS AND NRAS WILD TYPE METASTATIC COLORECTAL CANCER
    Medical condition: Colorectal Cancer
    Disease: Version SOC Term Classification Code Term Level
    17.0 10018065 - General disorders and administration site conditions 10069755 K-ras gene mutation PT
    17.0 10018065 - General disorders and administration site conditions 10071972 N-ras gene mutation PT
    17.0 100000004864 10052362 Metastatic colorectal cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) IT (Prematurely Ended) ES (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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