- Trials with a EudraCT protocol (509)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
509 result(s) found for: Beta Cell.
Displaying page 1 of 26.
EudraCT Number: 2009-014136-37 | Sponsor Protocol Number: Thal-002 | Start Date*: 2010-07-13 | |||||||||||||||||||||
Sponsor Name:University of Washington | |||||||||||||||||||||||
Full Title: A pilot study on the safety and efficacy of haemopoietic stem cell mobilization (CD34+ cells) with MOZOBIL ± G-CSF, in adult patients diagnosed with beta-thalassaemia major. | |||||||||||||||||||||||
Medical condition: Beta-thalassaemia major | |||||||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GR (Ongoing) | |||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-000208-13 | Sponsor Protocol Number: ACE-536-B-THAL-004 | Start Date*: 2019-12-09 | |||||||||||
Sponsor Name:Celgene Corporation | |||||||||||||
Full Title: A phase 2a study to evaluate the safety and pharmacokinetics of Luspatercept (ACE-536) in paediatric participants who require regular red blood cell transfusions due to beta (β) thalassemia | |||||||||||||
Medical condition: Beta-Thalassemia | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) GR (Trial now transitioned) IT (Restarted) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2016-003611-35 | Sponsor Protocol Number: HGB-212 | Start Date*: 2017-07-24 | |||||||||||
Sponsor Name:bluebird bio, Inc. | |||||||||||||
Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex V... | |||||||||||||
Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥ 8 transfusions of pRBCs per year... | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) GR (Completed) FR (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2014-001936-12 | Sponsor Protocol Number: MD2014.01 | Start Date*: 2019-02-11 | ||||||||||||||||
Sponsor Name:Sanquin Plasma Products BV | ||||||||||||||||||
Full Title: Efficacy and Safety of human apotransferrin in patients with β-thalassemia | ||||||||||||||||||
Medical condition: Beta thalassemia intermedia | ||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: NL (Prematurely Ended) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2015-004122-33 | Sponsor Protocol Number: HGB-207 | Start Date*: 2016-11-10 | |||||||||||
Sponsor Name:bluebird bio, Inc. | |||||||||||||
Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who do not have β0/β0 Genotype, by Transplantation of Autologous ... | |||||||||||||
Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, who do not have beta0/beta0 genotype, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) ... | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) DE (Completed) GR (Completed) GB (GB - no longer in EU/EEA) FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-006390-37 | Sponsor Protocol Number: VX21-CTX001-161 | Start Date*: Information not available in EudraCT | ||||||||||||||||
Sponsor Name:Vertex Pharmaceuticals Incorporated | ||||||||||||||||||
Full Title: A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Depend... | ||||||||||||||||||
Medical condition: Transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) | ||||||||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: DE (Ongoing) IT (Ongoing) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-002172-39 | Sponsor Protocol Number: VX21-CTX001-141 | Start Date*: 2021-10-05 | |||||||||||
Sponsor Name:VERTEX PHARMACEUTICALS INCORPORATED | |||||||||||||
Full Title: A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thalassemia | |||||||||||||
Medical condition: Transfusion-Dependent ß Thalassemia | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Ongoing) DE (Temporarily Halted) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-002499-15 | Sponsor Protocol Number: A536-04 | Start Date*: 2012-12-19 | ||||||||||||||||
Sponsor Name:ACCELERON PHARMA INC | ||||||||||||||||||
Full Title: A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients with β-Thalassemia Intermedia | ||||||||||||||||||
Medical condition: β-thalassemia intermedia | ||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: IT (Completed) GR (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2018-002935-88 | Sponsor Protocol Number: VX18-CTX001-131 | Start Date*: 2019-03-13 | ||||||||||||||||
Sponsor Name:Vertex Pharmaceuticals Incorporated | ||||||||||||||||||
Full Title: A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001) | ||||||||||||||||||
Medical condition: Transfusion-dependent β thalassemia (TDT). Severe sickle cell disease (SCD). | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: DE (Ongoing) GB (GB - no longer in EU/EEA) BE (Ongoing) FR (Ongoing) GR (Prematurely Ended) IT (Ongoing) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-001852-41 | Sponsor Protocol Number: GLP1-FMT | Start Date*: 2022-09-06 |
Sponsor Name:Radboud university medical center | ||
Full Title: BetaFIT study: Beta cell imaging after Faecal mIcrobiota Transplantation | ||
Medical condition: Type 1 diabetes | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: NL (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2014-004860-39 | Sponsor Protocol Number: TIGET-BTHAL | Start Date*: 2015-03-04 | |||||||||||
Sponsor Name:OSPEDALE SAN RAFFAELE | |||||||||||||
Full Title: A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment ... | |||||||||||||
Medical condition: Beta talassemia trasfusione dipendente | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-002959-17 | Sponsor Protocol Number: CR-BD-001 | Start Date*: 2016-12-02 | |||||||||||
Sponsor Name:Kiadis Pharma Netherlands B.V. | |||||||||||||
Full Title: An exploratory, open-label study to evaluate the safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic t... | |||||||||||||
Medical condition: Patients with beta-thalassemia major who are eligible for a haploidentical HSCT | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2012-000695-42 | Sponsor Protocol Number: HGB-205 | Start Date*: 2012-12-31 | |||||||||||
Sponsor Name:bluebird bio, Inc. | |||||||||||||
Full Title: A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem... | |||||||||||||
Medical condition: Severe sickle cell anemia and transfusion-dependent beta-thalassemia major. This study will enroll patients who are eligible for an allogeneic hematopoietic stem cell transplant (HSCT) but do not... | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-003351-38 | Sponsor Protocol Number: CTX001-111 | Start Date*: 2018-07-25 | |||||||||||
Sponsor Name:Vertex Pharmaceuticals Incorporated | |||||||||||||
Full Title: A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion-Dependen... | |||||||||||||
Medical condition: Transfusion-Dependent β Thalassemia | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Ongoing) IT (Ongoing) GB (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-005450-30 | Sponsor Protocol Number: ABI-MS-P01 | Start Date*: 2014-01-08 |
Sponsor Name:Medizinische Universität Innsbruck | ||
Full Title: Anti-Biopharmaceutical Immunization: Prediction and analysis of clinical relevance to minimize the risk of immunization in multiple sclerosis patients on interferon-beta treatment | ||
Medical condition: Development of neutralising antibodies against Interferon-beta in the treatment of multiple sclerosis | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: AT (Completed) SE (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2019-002989-12 | Sponsor Protocol Number: IMR-BTL-201 | Start Date*: 2020-06-02 | |||||||||||
Sponsor Name:IMARA, Inc. | |||||||||||||
Full Title: A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia | |||||||||||||
Medical condition: β-thalassemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DK (Prematurely Ended) NL (Prematurely Ended) FR (Ongoing) GR (Prematurely Ended) IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-005659-15 | Sponsor Protocol Number: ACE-011-B-THAL-001 | Start Date*: 2012-10-04 | |||||||||||||||||||||
Sponsor Name:Celgene Corporation | |||||||||||||||||||||||
Full Title: A Phase 2a, open-label, dose finding study to determine the safety and tolerability of sotatercept (ACE-011) in adults with beta (β)- thalassemia | |||||||||||||||||||||||
Medical condition: Subjects with red blood cell (RBC) transfusion dependent β-thalassemia major and β thalassemia intermedia, as well as non-transfusion dependent β-thalassemia intermedia will take part in this study. | |||||||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) IT (Prematurely Ended) GR (Completed) | |||||||||||||||||||||||
Trial results: View results |
EudraCT Number: 2020-005229-95 | Sponsor Protocol Number: PBKR03-001 | Start Date*: 2021-08-23 | |||||||||||
Sponsor Name:Passage Bio, Inc. | |||||||||||||
Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Dis... | |||||||||||||
Medical condition: Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactoce... | |||||||||||||
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Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
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 (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-002230-37 | Sponsor Protocol Number: CLAF237ANL05T | Start Date*: 2013-12-19 |
Sponsor Name:Erasmus Medical Center | ||
Full Title: “Novel Approach to Detect the Detailed Effects of Vildagliptin on Beta-cell Dynamics in Patients with Early-onset Type 2 Diabetes” | ||
Medical condition: impaired fasting glucose/impaired glucose tolerance, prediabetes | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: NL (Ongoing) | ||
Trial results: (No results available) |
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