284. Diamond-Blackfan anemia Clinical trials / Disease details
Clinical trials : 37 / Drugs : 110 - (DrugBank : 34) / Drug target genes : 23 - Drug target pathways : 124
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2011-006322-25-GB (EUCTR) | 08/10/2012 | 14/08/2012 | Extending the treatment of Iron Overload | A 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 LLC | NULL | Not Recruiting | Female: yes Male: yes | 1500 | Phase 2 | Egypt;United States;Canada;Thailand;Lebanon;Turkey;Italy;United Kingdom | ||
| 2 | EUCTR2011-006322-25-IT (EUCTR) | 20/08/2012 | 03/09/2012 | Extension to the treatment of iron overload with chelation therapy | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 1500 | Phase 3 | United States;United Kingdom;Italy | ||
| 3 | EUCTR2011-005675-16-IT (EUCTR) | 15/05/2012 | 02/08/2012 | Treatment 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. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | United States;Canada;Lebanon;Turkey;Italy | ||
| 4 | EUCTR2010-019645-25-IT (EUCTR) | 04/08/2010 | 15/07/2010 | A 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 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United Kingdom;Italy | ||
| 5 | EUCTR2010-019645-25-GB (EUCTR) | 19/07/2010 | 16/06/2010 | A 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 LLC | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | United States;Thailand;Turkey;Italy;United Kingdom | ||
| 6 | EUCTR2007-000766-20-GB (EUCTR) | 17/07/2008 | 13/12/2007 | 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 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 AG | NULL | Not 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): no | Italy;United Kingdom | ||
| 7 | NCT00673608 (ClinicalTrials.gov) | November 2007 | 5/5/2008 | Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload | A 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 Overload | Drug: deferasirox | Novartis | NULL | Completed | 18 Years | N/A | All | 118 | Phase 4 | Australia |
| 8 | NCT00235391 (ClinicalTrials.gov) | October 2005 | 6/10/2005 | Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload | A 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 Chelators | Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Myelofibrosis | Drug: Deferasirox | Novartis Pharmaceuticals | NULL | Completed | 2 Years | N/A | All | 1683 | Phase 3 | United States;Belgium;Canada;Germany;Greece;Italy;Netherlands;Spain;Taiwan;Thailand;Turkey;United Kingdom;Australia |