286. Hereditary sideroblastic anemia
6 clinical trials,   13 drugs   (DrugBank: 5 drugs),   0 gene,   0 pathway

Searched query = "Hereditary sideroblastic anemia", "Congenital sideroblastic anemia", "Sideroblastic anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-UMIN000023490
2016/08/2005/08/2016The 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
2JPRN-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
3EUCTR2010-021062-29-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
4EUCTR2010-021062-29-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
5EUCTR2010-021062-29-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2007-000766-20-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%.Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
192Phase 2Italy;United Kingdom