286. Hereditary sideroblastic anemia Clinical trials / Disease details


Clinical trials : 7 Drugs : 20 - (DrugBank : 10) / Drug target genes : 8 - Drug target pathways : 43

  
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PhaseCountries
1NCT04965597
(ClinicalTrials.gov)
April 19, 202221/6/2021Treosulfan-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 DiseasesBone 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 SyndromeDrug: 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 AssessmentFred Hutchinson Cancer CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States
2JPRN-UMIN000023490
2016/08/2005/08/2016The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic AnemiaThe 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 MedicineNULLComplete: follow-up complete20years-old80years-oldMale and Female5Not applicableJapan
3JPRN-UMIN000021407
2016/03/1515/03/2016A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemiaA 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 anemiaOrally 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 UniversitySBI Pharmaceuticals Co., LtdComplete: follow-up continuing20years-old80years-oldMale and Female5Not applicableJapan
4EUCTR2010-021062-29-GR
(EUCTR)
12/04/201102/02/2011Phase 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) - HYPERIONPhase 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 AGNULLNot RecruitingFemale: yes
Male: yes
45Phase 2Greece;Italy;United Kingdom
5EUCTR2010-021062-29-IT
(EUCTR)
30/12/201029/11/2010Phase 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) - HYPERIONPhase 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 FARMANULLNot RecruitingFemale: yes
Male: yes
45Phase 2Greece;United Kingdom;Italy
6EUCTR2010-021062-29-GB
(EUCTR)
21/12/201020/10/2010Phase 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) - HYPERIONPhase 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 AGNULLNot RecruitingFemale: yes
Male: yes
45Phase 2Greece;Italy;United Kingdom
7EUCTR2007-000766-20-GB
(EUCTR)
17/07/200813/12/2007A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIAA 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 AGNULLNot 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): noItaly;United Kingdom