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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   44359   clinical trials with a EudraCT protocol, of which   7384   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 48 of 71.
    EudraCT Number: 2010-021430-64 Sponsor Protocol Number: RR10/9389 Start Date*: 2010-12-03
    Sponsor Name:University of Leeds
    Full Title: A randomised double blind placebo controlled clinical trial of anti B-cell therapy in patients with primary Sjögren’s syndrome.
    Medical condition: Primary Sjögren’s syndrome (PSS).
    Disease: Version SOC Term Classification Code Term Level
    14.1 10028395 - Musculoskeletal and connective tissue disorders 10040767 Sjogren's syndrome PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-001175-31 Sponsor Protocol Number: ALM201/0001 Start Date*: 2014-10-10
    Sponsor Name:Almac Discovery
    Full Title: A phase I open-label multicentre dose-escalation study of subcutaneous ALM201 in patients with advanced ovarian cancer and other solid tumours.
    Medical condition: Advanced ovarian cancer and other solid tumours
    Disease: Version SOC Term Classification Code Term Level
    17.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10049280 Solid tumour LLT
    17.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10033128 Ovarian cancer PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-005594-21 Sponsor Protocol Number: ADP-04511 Start Date*: 2016-08-18
    Sponsor Name:Adaptimmune LLC
    Full Title: A Pilot Study of Genetically Engineered NY-ESO-1 Specific NY-ESO-1c259T in HLA-A2+ Patients with Synovial Sarcoma
    Medical condition: Unresectable, metastatic or recurrent synovial sarcomas
    Disease: Version SOC Term Classification Code Term Level
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10042863 Synovial sarcoma PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-005863-29 Sponsor Protocol Number: HelmetHeparin Start Date*: 2021-07-15
    Sponsor Name:ASST FATEBENEFRATELLI SACCO
    Full Title: Nebulised heparin in COVID-19-related ARDS patients undergoing non-invasive ventilation with helmet cPAP: a prospective, randomised, double blind, placebo-controlled, multicentre study
    Medical condition: COVID-19-related Acute Respiratory Distress Syndrome (ARDS) patients undergoing non-invasive ventilation with helmet continuous positive airway pressure (cPAP)
    Disease: Version SOC Term Classification Code Term Level
    22.0 10042613 - Surgical and medical procedures 10052956 CPAP LLT
    23.1 10042613 - Surgical and medical procedures 10084460 COVID-19 treatment PT
    21.1 100000004855 10003083 ARDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-000907-19 Sponsor Protocol Number: ML29755 Start Date*: 2015-09-10
    Sponsor Name:University Medical Center Groningen
    Full Title: MPDL3280A treatment in patients with locally advanced or metastatic solid tumors after or during investigational imaging
    Medical condition: solid tumors
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-005197-35 Sponsor Protocol Number: S1404 Start Date*: 2017-06-06
    Sponsor Name:Irish Clinical Oncology Research Group CLG, trading as Cancer Trials Ireland
    Full Title: A Phase III Randomized Trial Comparing Physician/Patient Choice of Either High Dose Interferon or Ipilimumab to MK-3475 (Pembrolizumab) in Patients with High Risk Resected Melanoma.
    Medical condition: High risk resected melanoma.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-001189-98 Sponsor Protocol Number: NICSA Start Date*: 2017-08-28
    Sponsor Name:Aarhus Universitet
    Full Title: Neoadjuvant short-term Intensive Chemoresection versus Standard Adjuvant intravesical instillations in NMIBC - A study on effect and tolerability of neoadjuvant short-term intensive chemoresection...
    Medical condition: Patients with recurrent non-muscle invasive bladder cancer
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000017467 10005004 Bladder cancer NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2004-000944-26 Sponsor Protocol Number: HOVON 65 MM / GMMG-HD4 Start Date*: 2007-05-02
    Sponsor Name:HOVON foundation
    Full Title: A randomized phase III study on the effect of Bortezomib combined with Adriamycin, Dexamethasone (AD) for induction treatment, followed by High Dose Melphalan and Bortezomib alone during maintenanc...
    Medical condition: multiple myeloma
    Disease: Version SOC Term Classification Code Term Level
    8.1 10028228 Multiple myeloma LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-000860-32 Sponsor Protocol Number: UMCN-AKF-14.17 Start Date*: 2015-04-28
    Sponsor Name:Radboud University Medical Center
    Full Title: Personalizing enzalutamide (Xtandi®) therapy by understanding the relation between the decrease in the expression profile of a panel of preselected microRNAs, tumor related mRNAs and treatment resp...
    Medical condition: Metastatic Castration Resistant Prostate Cancer (mCRPC)
    Disease: Version SOC Term Classification Code Term Level
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036909 Prostate cancer metastatic PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10060862 Prostate cancer PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10062904 Hormone-refractory prostate cancer PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-003126-42 Sponsor Protocol Number: DT-DEC01-DMD-CT-Phase1/2 Start Date*: 2023-12-12
    Sponsor Name:Dystrogen Therapeutics Technology Polska Spółka z Ograniczoną Odpowiedzialnością
    Full Title: A Phase 1/2, Open-Label, Exploratory Clinical Trial to Evaluate the Safety and Efficacy of DT-DEC01 Therapy in Patients with Duchenne Muscular Dystrophy
    Medical condition: Duchenne muscular dystrophy (DMD) is the most common inherited neuromuscular disease that mostly affects boys. DMD is a type of X-linked disease caused by a mutation of the gene at the Xp21 locus t...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000245-39 Sponsor Protocol Number: BGB-A317-303 Start Date*: 2018-06-21
    Sponsor Name:BeiGene Ltd., c/o BeiGene USA Inc.
    Full Title: A Phase 3, Open-Label, Multicenter, Randomized Study to Investigate the Efficacy and Safety of BGB-A317 (Anti−PD1 Antibody) Compared with Docetaxel in Patients with Non−Small Cell Lung Cancer Who H...
    Medical condition: Previously treated Non−Small Cell Lung Cancer in the second or third line setting.
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SK (Completed) LT (Completed) BG (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2017-001203-79 Sponsor Protocol Number: IM011021 Start Date*: 2017-12-04
    Sponsor Name:Bristol-Myers Squibb international Corporation
    Full Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of BMS-986165 in Subjects with Systemic Lupus Erythematosus
    Medical condition: Systemic Lupus Erythematosus
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000017968 10042947 Systemic lupus erythematosus synd LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) PL (Completed) DE (Completed) ES (Ongoing) RO (Completed)
    Trial results: View results
    EudraCT Number: 2016-000602-10 Sponsor Protocol Number: SNT-III-012 Start Date*: 2016-11-17
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Ste...
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) SE (Prematurely Ended) GB (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Completed) IT (Prematurely Ended) IE (Completed) HU (Prematurely Ended) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003951-23 Sponsor Protocol Number: AG-221-AML-005 Start Date*: 2016-07-08
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 1b/2 Open-Label, Randomized Study of 2 Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine: Oral AG-120 Plus Subcutaneous Azacitidine and Oral AG-221 P...
    Medical condition: Newly diagnosed acute myeloid leukemia (AML) habouring an isocitrate dehydrogenase 1 (IDH1) or an isocitrate dehydrogenase 2 (IDH2) mutation, respectively, who are not candidates to receive intensi...
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10000886 Acute myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) PT (Completed) ES (Ongoing) NL (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-002749-23 Sponsor Protocol Number: AGMT_MM-1/EMN-13 Start Date*: 2015-01-19
    Sponsor Name:AGMT gGmbH
    Full Title: Ixazomib in combination to thalidomide - dexamethasone for patients with relapsed and/or refractory multiple myeloma
    Medical condition: refractory and/or relapsed multiple myeloma
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) CZ (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2010-023430-23 Sponsor Protocol Number: EMR701048_009 Start Date*: 2011-01-17
    Sponsor Name:Merck Serono S.A.
    Full Title: A phase IIIb, open-label, single-arm, multicenter study to assess the immunogenicity of the r-hGH liquid multidose formulation (Saizen solution for injection) when administered to male and female a...
    Medical condition: This is GH replacement therapy in adult subjects with documented Growth Hormone Deficiency (GHD) either idiopathic or acquired and childhood- or adult-onset
    Disease: Version SOC Term Classification Code Term Level
    12.1 10056438 Growth hormone deficiency LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HU (Completed) CZ (Completed) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-003248-10 Sponsor Protocol Number: bb2121-MM-007 Start Date*: 2021-02-09
    Sponsor Name:Celgene Corporation
    Full Title: An exploratory phase 1/2 trial to determine recommended phase 2 dose (RP2D), safety and preliminary efficacy of bb2121 (ide-cel) combinations in subjects with refractory/relapsed multiple myeloma
    Medical condition: Multiple myeloma (MM) with progression during or within 6 months of the last treatment.
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10028228 Multiple myeloma LLT
    21.1 100000004864 10067095 Multiple myeloma progression LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-005220-26 Sponsor Protocol Number: ACE-LY-308 Start Date*: 2017-05-16
    Sponsor Name:Acerta Pharma BV
    Full Title: A Phase 3, Randomized, Double blind, Placebo controlled, Multicenter Study of Bendamustine and Rituximab (BR) alone Versus in Combination with Acalabrutinib (ACP 196) in Subjects with Previously Un...
    Medical condition: Mantle Cell Lymphoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004851 10026798 Mantle cell lymphomas HLT
    Population Age: Elderly Gender: Male, Female
    Trial protocol: HU (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing) CZ (Trial now transitioned) GR (Trial now transitioned) PL (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) RO (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-001604-28 Sponsor Protocol Number: EMR200095-006 Start Date*: 2017-02-07
    Sponsor Name:Merck KGaA
    Full Title: A Phase Ib/II Multicenter, Randomized, Open Label Trial to Compare Tepotinib(MSC2156119J) Combined with Gefitinib Versus Chemotherapy as Second-line Treatment in Subjects with MET Positive, Locally...
    Medical condition: Locally Advanced or Metastatic 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) 10059515 Non-small cell lung cancer metastatic PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) SK (Ongoing) BG (Completed) NL (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-000600-29 Sponsor Protocol Number: UX023-CL301 Start Date*: 2016-09-15
    Sponsor Name:Ultragenyx Pharmaceuticals Inc
    Full Title: A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients with X-linked Hypophosphatemia (XLH)
    Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, defective bone mineralisation, and impaired growth plate or endochondral ossification caused by inactivating mutations in t...
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) IE (Completed) DE (Completed) ES (Prematurely Ended) SE (Completed) GB (Completed) IT (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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