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Clinical trials for Vector

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   44294   clinical trials with a EudraCT protocol, of which   7351   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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    250 result(s) found for: Vector. Displaying page 1 of 13.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2019-003555-11 Sponsor Protocol Number: PD20180302 Start Date*: 2021-04-19
    Sponsor Name:ASSISTANCE PUBLIQUE-HOPÏTAUX DE PARIS
    Full Title: A Phase 1/2 Open Label non randomized Study, multicentric, single arm evaluating the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the ...
    Medical condition: immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis)
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-000684-16 Sponsor Protocol Number: 03MI10 Start Date*: 2010-01-21
    Sponsor Name:Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health
    Full Title: Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector.
    Medical condition: X-Linked severe combined Immunodeficiency (SCID-X1)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010099 Combined immunodeficiency LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-001383-18 Sponsor Protocol Number: Start Date*: 2015-11-30
    Sponsor Name:University of Oxford
    Full Title: An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)
    Medical condition: Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the...
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004853 10008791 Choroideremia LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-004266-18 Sponsor Protocol Number: LTF-307 Start Date*: 2020-09-03
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
    Medical condition: Subjects with sickle-cell disease treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety an...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10040648 Sickle cell SC disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-005080-40 Sponsor Protocol Number: FLT180a-04 Start Date*: 2018-05-15
    Sponsor Name:Freeline Therapeutics Limited
    Full Title: An Open-Label, Multicentre, Long-Term Follow-Up Study to Investigate the Safety and Durability of Response Following Dosing of a Novel Adeno-Associated Viral Vector (FLT180a) in Patients with Haemo...
    Medical condition: Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018939 Haemophilia 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: 2016-000925-38 Sponsor Protocol Number: UCL13/0076 Start Date*: 2017-01-04
    Sponsor Name:University College London (UCL)
    Full Title: GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding Factor VIII-V3
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2011-006100-12 Sponsor Protocol Number: FANCOLEN-1 Start Date*: 2013-04-12
    Sponsor Name:FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS
    Full Title: Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with...
    Medical condition: Fanconi anemia (Subtype A)
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-024253-36 Sponsor Protocol Number: 10MI29 Start Date*: 2012-08-03
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1αS-ADA lentiviral vector mediated gene modification of autologus CD34+ cel...
    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
    20.0 100000012248 10066372 ADA deficiency LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-000206-28 Sponsor Protocol Number: SB-318-1502 Start Date*: 2018-08-14
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis I (MPS I)
    Medical condition: Mucopolysaccharidosis type I (MPS I)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2006-000985-34 Sponsor Protocol Number: 001:CD40L Start Date*: 2006-06-15
    Sponsor Name:Clinical Immunology Division
    Full Title: A Phase I-IIa Study of Dose escalating Intravesical AdCD40L instillation in Urinary Bladder Carcinoma
    Medical condition: Urinary bladder cancer is an attractive target for immunostimulating gene therapy because it is sensitive for immunotherapy as demonstrated by the life-prolonging effect of bacillus Calmette-Guérin...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-003431-29 Sponsor Protocol Number: MGT012 Start Date*: 2019-06-24
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/8-hG1.7p.coCNGA3) for gene therapy of children with achromatopsia owing to defects in CNGA3
    Medical condition: Achromatopsia caused by mutations in the CNGA3 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    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: 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: 2017-000852-24 Sponsor Protocol Number: 15/0552 Start Date*: 2017-08-23
    Sponsor Name:University College London (UCL)
    Full Title: A Phase I/II, Open label, Multicentre, Ascending Single Dose, Safety Study of a Novel Adeno-associated Viral Vector (FLT180a) in Patients With Haemophilia B
    Medical condition: Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018939 Haemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-000192-33 Sponsor Protocol Number: SB-913-1602 Start Date*: 2018-08-17
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II)
    Medical condition: Mucopolysaccharidosis type II (MPS II)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-002502-31 Sponsor Protocol Number: RP-L102-0118 Start Date*: 2018-10-31
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemi...
    Medical condition: Fanconi anemia (subtype A)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-004805-42 Sponsor Protocol Number: SB-FIX-1501 Start Date*: 2018-06-05
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy via Zinc Finger Nuclease (ZFN) mediated targeted integration of SB-FIX in Subjects...
    Medical condition: Severe Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018939 Haemophilia B (Factor IX) LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2014-000274-20 Sponsor Protocol Number: GNT-WAS-03 Start Date*: 2014-05-14
    Sponsor Name:Genethon
    Full Title: LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08)
    Medical condition: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopeni...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10047992 Wiskott-Aldrich syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-000662-29 Sponsor Protocol Number: 270-203 Start Date*: 2018-02-01
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-000359-26 Sponsor Protocol Number: ESTEVE-SANF-201 Start Date*: 2016-04-29
    Sponsor Name:Laboratorios del Dr. Esteve, S.A.
    Full Title: Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients wit...
    Medical condition: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is an inherited lysosomal storage disease caused by a specific lysosomal enzyme deficiency that leads to intracellular accumulation of the ...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
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    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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