- Trials with a EudraCT protocol (41)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
41 result(s) found for: Vector control.
Displaying page 1 of 3.
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: 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: 2020-001235-27 | Sponsor Protocol Number: AVXS-101-CL-101 | Start Date*: 2020-04-06 |
Sponsor Name:AveXis, Inc. | ||
Full Title: Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101 | ||
Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | ||
Disease: | ||
Population Age: Newborns, Infants and toddlers, Under 18 | Gender: Male, Female | |
Trial protocol: Outside EU/EEA | ||
Trial results: View results |
EudraCT Number: 2021-002693-10 | Sponsor Protocol Number: VAC3_SARS-CoV2_seroconversion_study | Start Date*: 2021-07-15 |
Sponsor Name:Medical University of Vienna, Department for Internal Medicine III, Division of Rheumatology | ||
Full Title: A Phase II Study to Evaluate Safety and Efficacy to a Third Vaccination in Immunocompromised Patients with Inadequate Humoral Response after Primary mRNA SARS-CoV-2 (Covid-19) Vaccination | ||
Medical condition: Vaccination against SARS-CoV-2 in patients with immunosuppressive therapy or immunodeficiencies | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: AT (Completed) | ||
Trial results: (No results available) |
EudraCT Number: 2020-001461-36 | Sponsor Protocol Number: CT-AMT-130-02 | Start Date*: 2021-06-30 | |||||||||||
Sponsor Name:uniQure biopharma B.V. | |||||||||||||
Full Title: A Phase Ib/II Randomised, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Ther... | |||||||||||||
Medical condition: Huntington Disease | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: PL (Trial now transitioned) DE (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-003104-42 | Sponsor Protocol Number: NSR-CHM-OS2(273CH201) | Start Date*: 2018-01-05 | ||||||||||||||||
Sponsor Name:NightstaRx Ltd (A Biogen Company) | ||||||||||||||||||
Full Title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Prot... | ||||||||||||||||||
Medical condition: Choroideremia (CHM) X-Linked Retinitis Pigmentosa (XLRP) | ||||||||||||||||||
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Population Age: Adults | Gender: Male | |||||||||||||||||
Trial protocol: DE (Trial now transitioned) GB (GB - no longer in EU/EEA) FI (Completed) FR (Trial now transitioned) DK (Completed) NL (Completed) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-002348-57 | Sponsor Protocol Number: 2021-002348-57 | Start Date*: 2021-05-30 |
Sponsor Name:Medical University of Vienna | ||
Full Title: A Randomized, Parallel Group, Single-Blind, Phase 2 Study to Evaluate the immune response of two classes of SARS-Cov-2 Vaccines employed as Third Vaccination in Patients under current Rituximab The... | ||
Medical condition: Vaccination against SARS-CoV-2 in patients with rituximab therapy | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: AT (Completed) | ||
Trial results: View results |
EudraCT Number: 2022-003407-15 | Sponsor Protocol Number: SRP-9001-104 | Start Date*: 2023-08-08 |
Sponsor Name:Sarepta Therapeutics, Inc | ||
Full Title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-exis... | ||
Medical condition: Duchenne muscular dystrophy | ||
Disease: | ||
Population Age: Children, Under 18 | Gender: Male | |
Trial protocol: ES (Temporarily Halted) | ||
Trial results: (No results available) |
EudraCT Number: 2005-003587-34 | Sponsor Protocol Number: IPR/01 | Start Date*: 2007-04-12 |
Sponsor Name:MolMed | ||
Full Title: A phase I-II study: infusion of donor lymphocytes transduced with the suicide gene HSV TK, after transplantation of allogeneic T-depleted stem cells from a haploidentical donor in patients with hae... | ||
Medical condition: Hematological malignancies at high risk of relapse based on disease progression or presence of negative prognostic factor, who have received a HCTfrom donor HLA mismatched (haploidentical) for 2 o... | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: GR (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2017-002213-60 | Sponsor Protocol Number: GX1001 | Start Date*: 2019-01-08 | ||||||||||||||||
Sponsor Name:Solid Biosciences Inc. | ||||||||||||||||||
Full Title: A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with D... | ||||||||||||||||||
Medical condition: Duchenne muscular dystrophy | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-002568-62 | Sponsor Protocol Number: AVB-PGRN-001 | Start Date*: 2023-03-13 |
Sponsor Name:AviadoBio Ltd | ||
Full Title: A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal... | ||
Medical condition: Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: FR (Ongoing) IT (Trial now transitioned) ES (Ongoing) SE (Trial now transitioned) BE (Trial now transitioned) | ||
Trial results: (No results available) |
EudraCT Number: 2014-005004-21 | Sponsor Protocol Number: THOR-TUE-01 | Start Date*: 2015-12-23 | |||||||||||
Sponsor Name:Universitätsklinikum Tübingen, STZ eyetrial am Department für Augenheilkunde | |||||||||||||
Full Title: THOR - Tübingen Choroideremia gene therapy trial open label Phase 2 clinical trial using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1) | |||||||||||||
Medical condition: Choroideremia (CHM) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: DE (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-001452-55 | Sponsor Protocol Number: 204852 | Start Date*: 2018-11-26 | |||||||||||
Sponsor Name:GlaxoSmithKline Biologicals | |||||||||||||
Full Title: A first-time-in human (FTIH), Phase I/II, randomized, multi-centric, single-blind, controlled dose-escalation study to evaluate the reactogenicity, safety, immunogenicity and efficacy of GSK Biolog... | |||||||||||||
Medical condition: Chronic hepatitis B | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2016-003705-34 | Sponsor Protocol Number: RDC-PDE6A-01 | Start Date*: 2019-07-02 |
Sponsor Name:Universitätsklinikum Tübingen | ||
Full Title: PIGMENT – PDE6A gene therapy for retinitis pigmentosa | ||
Medical condition: PDE6A-linked retinitis pigmentosa | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: DE (Trial now transitioned) | ||
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: 2017-002187-40 | Sponsor Protocol Number: GS-LHON-CLIN-05 | Start Date*: 2018-03-15 |
Sponsor Name:GENSIGHT BIOLOGICS | ||
Full Title: Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for U... | ||
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: BE (Completed) FR (Completed) GB (GB - no longer in EU/EEA) ES (Ongoing) NL (Ongoing) IT (Completed) | ||
Trial results: (No results available) |
EudraCT Number: 2017-002204-27 | Sponsor Protocol Number: GS030_CLIN_001 | Start Date*: 2017-12-22 |
Sponsor Name:GENSIGHT-BIOLOGICS | ||
Full Title: A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa | ||
Medical condition: Retinitis Pigmentosa | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) | ||
Trial results: (No results available) |
EudraCT Number: 2019-000667-24 | Sponsor Protocol Number: ST-920-201 | Start Date*: 2019-11-12 | |||||||||||
Sponsor Name:Sangamo Therapeutics, Inc. | |||||||||||||
Full Title: A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Dise... | |||||||||||||
Medical condition: Fabry Disease (X-linked lysosomal storage disease) | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2015-003418-25 | Sponsor Protocol Number: CTU/2014/120 | Start Date*: 2016-03-29 | |||||||||||
Sponsor Name:MeiraGTx UK II Ltd | |||||||||||||
Full Title: An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with D... | |||||||||||||
Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65 | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2022-003314-37 | Sponsor Protocol Number: CCM1862 | Start Date*: 2023-04-19 | |||||||||||
Sponsor Name:CENTRO CARDIOLOGICO SPA FONDAZIONE MONZINO | |||||||||||||
Full Title: Effects of combined diuretic therapy on exercise capacity, alveolus-capillary diffusion, and maintenance of euvolemia in chronic congestive heart failure, diuretic therapy compared between loop diu... | |||||||||||||
Medical condition: congestive heart failure | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
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