- Trials with a EudraCT protocol (29)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (14)
29 result(s) found for: Globin.
Displaying page 1 of 2.
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: 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: 2021-000212-34 | Sponsor Protocol Number: AG348-C-018 | Start Date*: 2021-10-11 | |||||||||||
Sponsor Name:Agios Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZ... | |||||||||||||
Medical condition: Transfusion-Dependent Alpha- or Beta-Thalassemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Ongoing) DK (Trial now transitioned) IT (Trial now transitioned) BG (Trial now transitioned) FR (Trial now transitioned) NL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-000211-23 | Sponsor Protocol Number: AG348-C-017 | Start Date*: 2021-10-15 | |||||||||||
Sponsor Name:Agios Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non Transfusion-Dependent Alpha- or Beta-Thalassemia (ENE... | |||||||||||||
Medical condition: Non–Transfusion-Dependent Alpha- or Beta-Thalassemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Ongoing) DK (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned) BG (Trial now transitioned) NL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2020-005736-30 | Sponsor Protocol Number: CDAII-LUSPA_FA02-2020 | Start Date*: 2023-07-17 | ||||||||||||||||
Sponsor Name:FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia | ||||||||||||||||||
Full Title: A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll). | ||||||||||||||||||
Medical condition: Congenital Dyserythropoietic Anemia type II (CDAII) | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: IT (Prematurely Ended) | ||||||||||||||||||
Trial results: (No results available) |
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 (Completed) | |||||||||||||||||||||||
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 (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-001600-11 | Sponsor Protocol Number: MP4CO-13-SCD-206 | Start Date*: 2013-09-25 |
Sponsor Name:Sangart, Inc. | ||
Full Title: A Phase 2 Multi-center, Randomized, Double-blind, Comparator-Controlled Dose Finding Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects with Sickle Cell Disease | ||
Medical condition: Sickle cell disease (SCD) is an autosomal recessive disorder of the β globin gene of the hemoglobin molecule. One single point mutation results in a lifetime of chronic morbidity and mortality. Vas... | ||
Disease: | ||
Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |
Trial protocol: GB (Prematurely Ended) BE (Prematurely Ended) | ||
Trial results: (No results available) |
EudraCT Number: 2019-000331-63 | Sponsor Protocol Number: HGB-210 | Start Date*: 2021-02-09 | |||||||||||
Sponsor Name: bluebird bio, Inc. | |||||||||||||
Full Title: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease. | |||||||||||||
Medical condition: Sickle Cell Disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Prematurely Ended) GB (GB - no longer in EU/EEA) NL (Ongoing) IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-002395-36 | Sponsor Protocol Number: P-SP420-THAL-01 | Start Date*: 2022-12-16 | |||||||||||
Sponsor Name:Pharmacosmos A/S | |||||||||||||
Full Title: An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent β-thalassemia | |||||||||||||
Medical condition: Transfusion-dependent β-thalassemia | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Trial now transitioned) GR (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-003572-23 | Sponsor Protocol Number: GCP#01.01.030 | Start Date*: 2017-12-27 | ||||||||||||||||
Sponsor Name:Gamida Cell Ltd | ||||||||||||||||||
Full Title: Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies | ||||||||||||||||||
Medical condition: Hemoglobinopathies | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: FR (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2014-005115-16 | Sponsor Protocol Number: FORMA-04 | Start Date*: 2020-04-22 |
Sponsor Name:Octapharma AG | ||
Full Title: Prospective, open-label, uncontrolled, Phase III study to assess the efficacy, safety, and pharmacokinetics of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during an... | ||
Medical condition: Congenital fibrinogendeficiency | ||
Disease: | ||
Population Age: Newborns, Infants and toddlers, Children, Under 18 | Gender: Male, Female | |
Trial protocol: Outside EU/EEA | ||
Trial results: View results |
EudraCT Number: 2011-000973-30 | Sponsor Protocol Number: AMD-THAL | Start Date*: 2011-04-28 | |||||||||||
Sponsor Name:FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | |||||||||||||
Full Title: PLERIXAFOR MOBILIZED STEM CELLS AS SOURCE FOR GENE THERAPY OF BETA-THALASSEMIA AMD-THAL . | |||||||||||||
Medical condition: transfusion dependent beta thalassemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-001942-33 | Sponsor Protocol Number: CT2-02-17 | Start Date*: 2019-01-23 | |||||||||||
Sponsor Name:Rare Partners s.r.l. Impresa Sociale | |||||||||||||
Full Title: A personalized medicine approach for beta-thalassemia transfusion dependent patients: testing SIROLIMUS in a first pilot clinical trial. | |||||||||||||
Medical condition: beta-thalassemic patients | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-003505-96 | Sponsor Protocol Number: ISIS702843-CS2 | Start Date*: 2020-01-21 | |||||||||||
Sponsor Name:Ionis Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients with Non-Transf... | |||||||||||||
Medical condition: Non-Transfusion Dependent β-Thalassemia Intermedia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GR (Completed) | |||||||||||||
Trial results: View results |
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: 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: 2019-003489-41 | Sponsor Protocol Number: CADPT03A12101 | Start Date*: 2021-02-24 | |||||||||||
Sponsor Name:NOVARTIS PHARMA AG | |||||||||||||
Full Title: A first-in-patient Phase I/II clinical study to investigate the safety and efficacy of genome-edited hematopoietic stem and progenitor cells in subjects with severe complications of sickle cell dis... | |||||||||||||
Medical condition: Sickle Cell Disease (SCD) | |||||||||||||
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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: 2018-001469-18 | Sponsor Protocol Number: CT2-04-17 | Start Date*: 2019-04-17 | ||||||||||||||||
Sponsor Name:Dipartimento di Scienze della Vita e Biotecnologie dell'Università degli Studi di Ferrara | ||||||||||||||||||
Full Title: Treatment of beta-thalassemia patients with rapamycin (sirolimus): from pre-clinical research to a clinical trial | ||||||||||||||||||
Medical condition: Beta-thalassemia transfusion dependent patients | ||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: IT (Completed) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2011-003121-94 | Sponsor Protocol Number: P-Monofer-IBD-02 | Start Date*: 2011-12-14 | |||||||||||
Sponsor Name:Pharmacosmos A/S | |||||||||||||
Full Title: A Prospective, Non-controlled, Safety Study of Intravenous Iron Isomaltoside 1000 (Monofer®) administered by a High Dosing Regimen in Subjects with Inflammatory Bowel Disease (PROMISE) | |||||||||||||
Medical condition: Inflammatory Bowel Disease (IBD) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Completed) SE (Completed) NL (Completed) | |||||||||||||
Trial results: View results |
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