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 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.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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Target_
size
PhaseCountries
1JPRN-UMIN0000234902016/08/202 April 2019The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic AnemiaSideroblastic 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 MedicineNot Recruiting20years-old80years-oldMale and Female5Not applicableJapan
2JPRN-UMIN0000214072016/03/157 October 2019A 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 anemiasideroblastic 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., LtdNot Recruiting20years-old80years-oldMale and Female5Not applicableJapan
3EUCTR2007-000766-20-GB17/07/200820 March 2012A 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 - CORDELIABeta-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 AGAuthorisedFemale: yes
Male: yes
192Phase 2United Kingdom;Italy