- Trials with a EudraCT protocol (351)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
351 result(s) found for: Gene frequency.
Displaying page 1 of 18.
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: 2007-004308-11 | Sponsor Protocol Number: GTG002.07 | Start Date*: 2010-01-11 | |||||||||||
Sponsor Name:Genethon | |||||||||||||
Full Title: PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME | |||||||||||||
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-thrombocytopenia,... | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
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: 2019-002343-14 | Sponsor Protocol Number: RAG1-2019-01 | Start Date*: 2020-09-25 |
Sponsor Name:Leiden University Medical Center | ||
Full Title: PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY | ||
Medical condition: Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1) | ||
Disease: | ||
Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | |
Trial protocol: NL (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned) IT (Trial now transitioned) | ||
Trial results: (No results available) |
EudraCT Number: 2018-000673-68 | Sponsor Protocol Number: 16IC17 | Start Date*: 2018-10-09 | |||||||||||
Sponsor Name:Great Ormond Street Hospital for Children NHS Trust | |||||||||||||
Full Title: Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan | |||||||||||||
Medical condition: Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both... | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-005036-28 | Sponsor Protocol Number: 2012/41 | Start Date*: 2013-04-12 | |||||||||||
Sponsor Name:CENTRE ANTOINE LACASSAGNE | |||||||||||||
Full Title: Pharmacogenetic study in castration-resistant prostate cancer patients treated with abiraterone acetate | |||||||||||||
Medical condition: Prostate cancer metastatic | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2012-000677-23 | Sponsor Protocol Number: CLCL161A2201 | Start Date*: 2012-10-24 |
Sponsor Name:Novartis Pharma Services AG | ||
Full Title: A Phase II, multi-center, open-label, neoadjuvant, randomized study of weekly paclitaxel with or without LCL161 in patients with triple negative breast cancer | ||
Medical condition: Triple negative breast cancer | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Female | |
Trial protocol: GB (Completed) ES (Completed) IE (Completed) IT (Completed) BE (Completed) CZ (Completed) DE (Completed) | ||
Trial results: View results |
EudraCT Number: 2005-003814-14 | Sponsor Protocol Number: ML19498 | Start Date*: 2006-03-13 |
Sponsor Name:Unidad Integral de Investigación en Oncología, S.L. | ||
Full Title: Pharmacodynamic study of Erlotinib (Tarceva) in patients with mestastatic or locally advanced Epidermoid carcinoma of the head and neck | ||
Medical condition: Mestastatic or locally advanced Epidermoid carcinoma of the head and neck | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: ES (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2014-003965-11 | Sponsor Protocol Number: CC-TT-IMA-14 | Start Date*: 2015-11-18 |
Sponsor Name:University Medical Center Utrecht | ||
Full Title: Targeted therapy with Imatinib for treatment of poor prognosis mesenchymal-type resectable colon cancer: a proof-of-concept study in the preoperative window period. | ||
Medical condition: colon cancer | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: NL (Ongoing) | ||
Trial results: (No results available) |
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: 2018-003612-45 | Sponsor Protocol Number: A9001502 | Start Date*: 2019-09-06 |
Sponsor Name:Pfizer Inc. | ||
Full Title: TREATMENT RESISTANCE FOLLOWING ANTI-CANCER THERAPIES (TRANSLATE) | ||
Medical condition: Cancer | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: AT (Prematurely Ended) ES (Prematurely Ended) | ||
Trial results: View results |
EudraCT Number: 2021-002855-12 | Sponsor Protocol Number: RM-493-034 | Start Date*: 2022-04-22 |
Sponsor Name:Rhythm Pharmaceuticals Inc. | ||
Full Title: A 2-Stage (Open-Label Run-in followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled, Phase 2 study of Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Rece... | ||
Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH) | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: FR (Completed) DE (Completed) ES (Ongoing) NL (Completed) GR (Completed) | ||
Trial results: View results |
EudraCT Number: 2018-002849-11 | Sponsor Protocol Number: FIRE-5 | Start Date*: 2019-06-17 | |||||||||||
Sponsor Name:Klinikum der Universität München, Ludwig-Maximilians-Universität München, represented by the managing medical director | |||||||||||||
Full Title: Optimal anti-EGFR Treatment of mCRC Patients with Low-Frequency RAS Mutation | |||||||||||||
Medical condition: RAS-mutant metastatic colorectal cancer | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-003500-40 | Sponsor Protocol Number: PQ-110-002 | Start Date*: 2019-04-08 | |||||||||||
Sponsor Name:ProQR Therapeutics IV B.V. | |||||||||||||
Full Title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of QR-110 in Subjects with Leber Congenital Amaurosis (LCA) due to the C.2991+1655a>G Mutation (P.... | |||||||||||||
Medical condition: Leber Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: BE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-004761-33 | Sponsor Protocol Number: CRO1881 | Start Date*: 2012-03-08 | |||||||||||
Sponsor Name:Imperial College | |||||||||||||
Full Title: A randomised double-blind placebo-controlled Phase 2B clinical trial of repeated application of gene therapy in patients with cystic fibrosis | |||||||||||||
Medical condition: Cystic fibrosis | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-002873-24 | Sponsor Protocol Number: RM-493-035 | Start Date*: 2022-05-16 |
Sponsor Name:Rhythm Pharmaceuticals Inc. | ||
Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melano... | ||
Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH) | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: FR (Trial now transitioned) ES (Ongoing) DE (Trial now transitioned) GR (Trial now transitioned) NL (Trial now transitioned) | ||
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: 2017-001366-14 | Sponsor Protocol Number: 207757 | Start Date*: 2017-09-14 | |||||||||||
Sponsor Name:GLAXOSMITHKLINE RESEARCH AND DEVELOPMENT | |||||||||||||
Full Title: A long-term safety and efficacy follow-on study in participants with transfusion dependent β-thalassemia who have previously received GSK2696277 (autologous hematopoietic stem cells genetically mod... | |||||||||||||
Medical condition: Beta-thalassemia | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-002443-26 | Sponsor Protocol Number: OVG2012/04 | Start Date*: 2012-08-17 |
Sponsor Name:University of Oxford | ||
Full Title: A phase II, multi-centre, open labelled randomised control trial to describe immune & transcriptomic responses to trivalent inactivated vaccine (TIV) & MF59 adjuvanted influenza vaccine (ATIV) in 1... | ||
Medical condition: Healthy children will be immunised with two vaccines against influenza: Agrippal or Imuvac, or Fluad to study the immune responses to immunisation at a genetic level. | ||
Disease: | ||
Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | |
Trial protocol: GB (Completed) | ||
Trial results: View results |
EudraCT Number: 2012-001725-26 | Sponsor Protocol Number: X-CGD-Version2 | Start Date*: 2013-03-12 | |||||||||||
Sponsor Name:Johann Wolfgang Goethe-University | |||||||||||||
Full Title: A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector | |||||||||||||
Medical condition: Chronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to... | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) | |||||||||||||
Trial results: View results |
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