- Trials with a EudraCT protocol (250)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (1)
250 result(s) found for: Vector.
Displaying page 1 of 13.
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) | |||||||||||||
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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... | |||||||||||||
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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... | |||||||||||||
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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 | |||||||||||||
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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 | ||||||||||||||||||
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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) | |||||||||||||
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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... | |||||||||||||
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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) | |||||||||||||
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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 | |||||||||||||
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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 | |||||||||||||
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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) | |||||||||||||
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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) | |||||||||||||
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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 | |||||||||||||
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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... | |||||||||||||
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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 | |||||||||||||
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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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