284. Diamond-Blackfan anemia Clinical trials / Disease details


Clinical trials : 37 Drugs : 110 - (DrugBank : 34) / Drug target genes : 23 - Drug target pathways : 124

  
8 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2011-006322-25-GB
(EUCTR)
08/10/201214/08/2012Extending the treatment of Iron OverloadA Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602). Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, beta-thalassemia and Diamond-Blackfan anemia; aquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure.
MedDRA version: 16.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: SSP-004184AQ 50 mg Capsule
Product Code: SSP-004184AQ 50 mg
INN or Proposed INN: deferitazole
Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184AQ 100 mg Capsule
Product Code: SSP-004184AQ 100 mg
INN or Proposed INN: deferitazole
Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184AQ 200 mg Capsule
Product Code: SSP-004184AQ 200 mg
INN or Proposed INN: deferitazole
Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184AQ 250 mg Capsule
Product Code: SSP-004184AQ 250 mg
INN or Proposed INN: deferitazole
Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184AQ 375 mg Capsule
Product Code: SSP-004184AQ 375 mg
INN or Proposed INN: deferitazole
Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-m
Shire Development LLCNULLNot RecruitingFemale: yes
Male: yes
1500Phase 2Egypt;United States;Canada;Thailand;Lebanon;Turkey;Italy;United Kingdom
2EUCTR2011-006322-25-IT
(EUCTR)
20/08/201203/09/2012Extension to the treatment of iron overload with chelation therapyA PHASE 3, OPEN-LABEL, MILTICENTRE, EXTENSION SAFETY AND TOLERABILITY STUDY FOR TRANSFUSIONALLY IRON OVERLOADED CHILDRE, ADOLISCENTS AND ADULTS USING FBS0701 (SSP-004184) - SPD602-301 (FBS0701 - CTP - 15) ADULT PATIENTS SUFFERING FROM IRON OVERLOAD OF TRANSFUSION-DEPENDENT DOCUMENTED, IN WHICH THE FOLLOWING PRIMARY DIAGNOSIS: ANEMIA HEREDITARY (EG sickle cell anemia), thalassemia and Diamond-Blackfan anemia
MedDRA version: 15.0;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: FBS0701 CAPSULE
Product Code: FBS0701/SSP004184
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701/SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701/SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701/SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701/SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
FERROKIN BIOSCIENCES INC.NULLNot RecruitingFemale: yes
Male: yes
1500Phase 3United States;United Kingdom;Italy
3EUCTR2011-005675-16-IT
(EUCTR)
15/05/201202/08/2012Treatment of chronic iron overload with a chelation therapy (FBS0701)A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload Patients with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),ß-thalassemia and Diamond Blackfan anemia;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: Iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: Iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents
FERROKIN BIOSCIENCES INC.NULLNot RecruitingFemale: yes
Male: yes
50Phase 2United States;Canada;Lebanon;Turkey;Italy
4EUCTR2010-019645-25-IT
(EUCTR)
04/08/201015/07/2010A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy. - NDA Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy. - ND Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure.Product Name: FBS0701 Capsule
Product Code: FBS0701
Product Name: FBS0701 Capsule
Product Code: FBS0701
Product Name: FBS0701 Capsule
Product Code: FBS0701
Product Name: FBS0701 Capsule
Product Code: FBS0701
FERROKIN BIOSCIENCES INC.NULLNot RecruitingFemale: yes
Male: yes
40Phase 2United Kingdom;Italy
5EUCTR2010-019645-25-GB
(EUCTR)
19/07/201016/06/2010A 24 week trial to study the safety and tolerability of SSP-004184 in the treatment of patients with longterm iron overload who need iron chelation therapy, with the option of 72 weeks further dosing.A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, with a 72 Week Dosing Extension. - SPD602-201 Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure.
MedDRA version: 14.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: SSP-004184 50 mg Capsule
Product Code: SSP-004184 50 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 100 mg Capsule
Product Code: SSP-004184 100 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 250 mg Capsule
Product Code: SSP-004184 250 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 375 mg Capsule
Product Code: SSP-004184 375 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 500 mg Capsule
Product Code: SSP-004184 500 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo
Shire Development LLCNULLNot RecruitingFemale: yes
Male: yes
50Phase 2United States;Thailand;Turkey;Italy;United Kingdom
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%.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
7NCT00673608
(ClinicalTrials.gov)
November 20075/5/2008Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron OverloadA Study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in Patients With Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or Other Anaemias Treated With Exjade® (Deferasirox) (The MILE Study)Hemoglobinopathies;Myelodysplastic Syndromes;Other Inherited or Acquired Anaemia;MPD Syndrome;Diamond-Blackfan Anemia;Other Rare Anaemias;Transfusional Iron OverloadDrug: deferasiroxNovartisNULLCompleted18 YearsN/AAll118Phase 4Australia
8NCT00235391
(ClinicalTrials.gov)
October 20056/10/2005Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron OverloadA Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron ChelatorsThalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;MyelofibrosisDrug: DeferasiroxNovartis PharmaceuticalsNULLCompleted2 YearsN/AAll1683Phase 3United States;Belgium;Canada;Germany;Greece;Italy;Netherlands;Spain;Taiwan;Thailand;Turkey;United Kingdom;Australia