286. Hereditary sideroblastic anemia
[
3 clinical trials,
7 drugs（DrugBank:
4 drugs),
0 target gene / 0 target pathway ]
Searched query = "Hereditary sideroblastic anemia", "Congenital sideroblastic anemia", "Sideroblastic anemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000023490 | 2016/08/20 | 2 April 2019 | 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 | Not Recruiting | 20years-old | 80years-old | Male and Female | 5 | Not applicable | Japan | ||
| 2 | JPRN-UMIN000021407 | 2016/03/15 | 7 October 2019 | 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 | Not Recruiting | 20years-old | 80years-old | Male and Female | 5 | Not applicable | Japan |
| 3 | EUCTR2007-000766-20-GB | 17/07/2008 | 20 March 2012 | 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 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Trade Name: Desferal Product Name: Desferal Product Code: DFO Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: deferoxamine CAS Number: 70-51-9 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Trade Name: Desferal Product Name: Desferal Product Code: DFO Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: deferoxamine CAS Number: 70-51-9 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2000- Trade Name: Exjade Product Name: Exjade Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Concentration unit: mg milligram(s) | Novartis Pharma Services AG | Authorised | Female: yes Male: yes | 192 | Phase 2 | United Kingdom;Italy |