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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 6 of 71.
    «« First « Previous 2  3  4  5  6  7  8  9  10  Next» Last»»
    EudraCT Number: 2017-000876-27 Sponsor Protocol Number: ATX-MTM-002 Start Date*: 2017-12-06
    Sponsor Name:Audentes Therapeutics Inc.
    Full Title: ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-L...
    Medical condition: X-linked Myotubular Myopathy (XLMTM)
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) FR (Trial now transitioned)
    Trial results: View results
    EudraCT Number: 2018-004467-31 Sponsor Protocol Number: DECENDO Start Date*: 2019-04-05
    Sponsor Name:
    Full Title: An exploratory study: Dendritic cells for immunotherapy of metastatic endometrial cancer patients
    Medical condition: metastatic endometrial cancer
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2012-000242-35 Sponsor Protocol Number: G1XCGD.01 Start Date*: 2013-01-10
    Sponsor Name:Genethon
    Full Title: A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease
    Medical condition: X-linked Chronic Granulomatous Disease
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10008906 Chronic granulomatous disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-002696-10 Sponsor Protocol Number: CCPK850X2202 Start Date*: 2018-05-09
    Sponsor Name:Novartis Pharma AG
    Full Title: An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa c...
    Medical condition: retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-000562-36 Sponsor Protocol Number: BIG2-06/N063D/EGF106708 Start Date*: 2007-05-04
    Sponsor Name:Novartis Pharma Services AG
    Full Title: ALTTO (Adjuvant Lapatinib and/or Trastuzumab Treatment Optimisation) study. A randomised, multi-centre, open-label, phase III study of adjuvant lapatinib, trastuzumab, their sequence and their comb...
    Medical condition: Operable primary breast cancer with over expression/ amplification of HER2.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10006187 Breast cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) HU (Completed) FR (Completed) GB (Completed) SK (Completed) EE (Completed) CZ (Completed) DK (Completed) BE (Completed) DE (Completed) GR (Completed) NL (Completed) IT (Completed) ES (Completed) SI (Completed) BG (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2007-001283-73 Sponsor Protocol Number: 109493 Start Date*: 2007-08-16
    Sponsor Name:GlaxoSmithKline Biologicals
    Full Title: A double-blind, randomized, placebo-controlled Phase III study to assess the efficacy of recMAGE-A3 + AS15 Antigen-Specific Cancer Immunotherapeutic as adjuvant therapy in patients with resectable ...
    Medical condition: Adjuvant therapy in patients with MAGE-A3-positive Non-Small Cell Lung Cancer (NSCLC) and who have had complete surgical resection
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029520 Non-small cell lung cancer stage IIIA PT
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029517 Non-small cell lung cancer stage I PT
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029518 Non-small cell lung cancer stage II PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) DE (Prematurely Ended) IE (Prematurely Ended) FI (Prematurely Ended) ES (Prematurely Ended) SI (Prematurely Ended) FR (Completed) SE (Prematurely Ended) GR (Prematurely Ended) IT (Prematurely Ended) LV (Prematurely Ended) AT (Completed) NL (Prematurely Ended) HU (Prematurely Ended) CZ (Prematurely Ended) EE (Prematurely Ended) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2006-001571-37 Sponsor Protocol Number: AAV2/2-hRPE65p-hRPE65 Start Date*: 2006-12-14
    Sponsor Name:University College London
    Full Title: AN OPEN-LABEL DOSE ESCALATION STUDY OF AN ADENO-ASSOCIATED VIRUS VECTOR (AAV2/2-hRPE65p-hRPE65) FOR GENE THERAPY OF SEVERE EARLY-ONSET RETINAL DEGENERATION
    Medical condition: The condition to be investigated is severe early-onset inherited retinal degeneration due to defects in the gene encoding RPE65
    Disease: Version SOC Term Classification Code Term Level
    13.1 10015919 - Eye disorders 10038845 Retinal degeneration PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-002979-34 Sponsor Protocol Number: LTFU-ABO-102 Start Date*: 2020-05-18
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
    Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progress...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005200-19 Sponsor Protocol Number: I-CONIC Start Date*: 2022-05-09
    Sponsor Name:Västra Götalandsregionen/Onkologiska kliniken
    Full Title: Use of Imatinib to convert triple negative breast cancer into ER-positive breast cancer - "I-CONIC"
    Medical condition: Early Triple Negative Breast Cancer planned for surgery.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002548-21 Sponsor Protocol Number: ProgettoAIRCId.21939 Start Date*: 2021-01-12
    Sponsor Name:FONDAZIONE IRCCS "ISTITUTO NAZIONALE DEI TUMORI"
    Full Title: Personalized therapy of metastatic thyroid cancer: biological characterization and optimization with 124I PET dosimetry
    Medical condition: metastatic thyroid cancer
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10076603 Poorly differentiated thyroid carcinoma PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-001275-23 Sponsor Protocol Number: OTL-101-5(17IC04) Start Date*: 2017-09-21
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Co...
    Medical condition: Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protect...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10066372 ADA deficiency LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2014-002392-28 Sponsor Protocol Number: RM-493-Supplementation-Therapy Start Date*: 2014-11-28
    Sponsor Name:Charité Univeritaetsmedizin Berlin
    Full Title: Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity
    Medical condition: Body weight is regulated within the hypothalamus. In rare cases mutations in genes, which are embedded in the signaling cascades of the hypothalamus lead to early onset severe obesity. POMC is one ...
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2007-000281-21 Sponsor Protocol Number: H6Q-MC-S039 Start Date*: 2007-09-05
    Sponsor Name:Eli Lilly and Company
    Full Title: Enzastaurin (LY317615) Before and Concomitant with Radiation Therapy, Followed by Enzastaurin Maintenance Therapy in Patients with Newly Diagnosed Glioblastoma without Methylation of the Promoter G...
    Medical condition: primary glioma without methylation of the promoter gene of MGMT enzyme
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10018336 Glioblastoma PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2021-000588-53 Sponsor Protocol Number: SNIPER Start Date*: 2023-02-07
    Sponsor Name:RIGENERAND Srl
    Full Title: RR001 in combination with chemotherapy for patients with locally advanced pancreatic adenocarcinoma: open-label, non-randomized dose escalation phase I/IIa study
    Medical condition: Locally advanced pancreatic adenocarcinoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10073364 Ductal adenocarcinoma of pancreas PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-021336-33 Sponsor Protocol Number: A8081014 Start Date*: 2010-12-30
    Sponsor Name:Pfizer Inc 235 East 42nd Street, New York, NY10017 US
    Full Title: PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF THE EFFICACY AND SAFETY OF CRIZOTINIB VERSUS PEMETREXED/CISPLATIN OR PEMETREXED/CARBOPLATIN IN PREVIOUSLY UNTREATED PATIENTS WITH NON-SQUAMOUS CARCINOMA OF ...
    Medical condition: non-small cell lung cancer (NSCLC)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029519 Non-small cell lung cancer stage III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) FI (Completed) IE (Completed) AT (Completed) IT (Completed) GB (Completed) BE (Completed) NL (Completed) DK (Prematurely Ended) PT (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2017-004310-25 Sponsor Protocol Number: ARRAY-797-301 Start Date*: 2018-12-14
    Sponsor Name:Array BioPharma Inc.
    Full Title: A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 in Patients with Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
    Medical condition: Dilated Cardiomyopathy (DCM) with Lamin A/C protein (LMNA) Mutation
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004849 10056419 Dilated cardiomyopathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NO (Prematurely Ended) BE (Completed) ES (Prematurely Ended) NL (Prematurely Ended) IT (Prematurely Ended) DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-003192-28 Sponsor Protocol Number: SOG-MIE-2014-04 Start Date*: 2015-01-02
    Sponsor Name:SOGUG
    Full Title: Phase II multicenter study to analyze the predictive value of fusion gene TMPRSS2-ETS in response to enzalutamide in patients with metastatic CRPC no previously treated with chemotherapy
    Medical condition: Metastatic castration-resistant prostate cancer no previously treated with chemotherapy
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10062904 Hormone-refractory prostate cancer PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2015-003582-28 Sponsor Protocol Number: LOXO-TRK-15002 Start Date*: Information not available in EudraCT
    Sponsor Name:Bayer Consumer Care AG
    Full Title: A Phase 2 Basket Study of the Oral TRK Inhibitor larotrectinib in Subjects with NTRK Fusion-Positive Tumors
    Medical condition: Solid Tumors
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10065252 Solid tumor LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DK (Trial now transitioned) IE (Completed) ES (Trial now transitioned) PT (Trial now transitioned) FR (Trial now transitioned) DE (Trial now transitioned) SE (Trial now transitioned) BE (Completed) CZ (Completed) SK (Completed) NO (Completed) HU (Completed) PL (Completed) GR (Completed) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-002051-42 Sponsor Protocol Number: R119861 Start Date*: 2019-10-25
    Sponsor Name:University of Manchester
    Full Title: A phase I-II, study of autologous CD34+ haematopoietic stem cells transduced ex vivo with CD11b lentiviral vector encoding for human SGSH in patients with mucopolysaccharidosis type IIIA (MPS IIIA,...
    Medical condition: MPS IIIA, mucopolysaccharidosis type IIIA
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10028093 Mucopolysaccharidosis PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001878-28 Sponsor Protocol Number: 307-201 Start Date*: 2019-12-17
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects...
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10034872 Phenylketonuria PT
    Population Age: 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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