286. Hereditary sideroblastic anemia Clinical trials / Disease details
Clinical trials : 7 / Drugs : 20 - (DrugBank : 10) / Drug target genes : 8 - Drug target pathways : 43
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04965597 (ClinicalTrials.gov) | April 19, 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 2 | JPRN-UMIN000023490 | 2016/08/20 | 05/08/2016 | The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia | The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia - The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia | Sideroblastic anemia (congenital and acquired) | Food supplement containing 150 mg of ALA phosphate was given per day for 24 weeks. For the evaluation of efficacy and safety, medical examinations as well as urine and blood tests will be performed prior to administration of ALA, then at 2 and 4 weeks, and thereafter every month (up to 12 months). | Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine | NULL | Complete: follow-up complete | 20years-old | 80years-old | Male and Female | 5 | Not applicable | Japan |
| 3 | JPRN-UMIN000021407 | 2016/03/15 | 15/03/2016 | A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia | A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia - Efficacy and safety of 5-ALA phosphate in patients with sideroblastic anemia | sideroblastic anemia | Orally intake a 150 mg 5-aminolevulinic acid phosphate capsule once a day for up to 24 weeks. | Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University | SBI Pharmaceuticals Co., Ltd | Complete: follow-up continuing | 20years-old | 80years-old | Male and Female | 5 | Not applicable | Japan |
| 4 | EUCTR2010-021062-29-GR (EUCTR) | 12/04/2011 | 02/02/2011 | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | transfusion-dependent patients with TM, DBA or congenital sideroblastic anemia with severe cardiac iron overload MedDRA version: 13.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 10027433 - Metabolism and nutrition disorders | Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Trade Name: Desferal Product Name: deferoxamine Product Code: DFO INN or Proposed INN: DEFEROXAMINE MESILATE Trade Name: DESFERAL Product Name: deferoxamine Product Code: DFO INN or Proposed INN: DEFEROXAMINE MESILATE | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 2 | Greece;Italy;United Kingdom | ||
| 5 | EUCTR2010-021062-29-IT (EUCTR) | 30/12/2010 | 29/11/2010 | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | Transfusion-dependent patients with TM, DBA or congenital sideroblastic anemia with severe cardiac iron overload MedDRA version: 9.1;Level: LLT;Classification code 10043392 | Trade Name: EXJADE INN or Proposed INN: DEFERASIROX Trade Name: EXJADE INN or Proposed INN: DEFERASIROX Trade Name: EXJADE INN or Proposed INN: DEFERASIROX Trade Name: DESFERAL INN or Proposed INN: Deferoxamine Trade Name: DESFERAL INN or Proposed INN: Deferoxamine | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 2 | Greece;United Kingdom;Italy | ||
| 6 | EUCTR2010-021062-29-GB (EUCTR) | 21/12/2010 | 20/10/2010 | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION) - HYPERION | transfusion-dependent patients with TM, DBA or congenital sideroblastic anemia with severe cardiac iron overload MedDRA version: 14.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 10027433 - Metabolism and nutrition disorders | Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: DEFERASIROX Other descriptive name: Exjade Product Name: deferoxamine Product Code: DFO INN or Proposed INN: DEFEROXAMINE MESILATE Product Name: deferoxamine Product Code: DFO INN or Proposed INN: DEFEROXAMINE MESILATE Trade Name: Exjade Product Name: Exjade Product Code: ICL670 INN or Proposed INN: deferasirox INN or Proposed INN: deferasirox INN or Proposed INN: deferasirox | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 2 | Greece;Italy;United Kingdom | ||
| 7 | EUCTR2007-000766-20-GB (EUCTR) | 17/07/2008 | 13/12/2007 | A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIA | A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIA | Beta-thalassemia major (TM) or Diamond Blackfan anemia (DBA) or myelodysplastic syndromes (MDS) (low and INT-1 risk as per the IPSS for MDS) or sideroblastic anemia and myocardial iron overload as indicated by a T2* =6 but not greater than 20 ms, with no symptoms of cardiac dysfunction, and an MRI measured LVEF =56%. | Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 INN or Proposed INN: deferasirox Other descriptive name: EXJADE Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 INN or Proposed INN: deferasirox Other descriptive name: EXJADE Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 INN or Proposed INN: deferasirox Other descriptive name: EXJADE Trade Name: Desferal Product Name: Desferal Product Code: DFO INN or Proposed INN: deferoxamine Trade Name: Desferal Product Name: Desferal Product Code: DFO | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 192 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Italy;United Kingdom |