284. ダイアモンド・ブラックファン貧血
［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］
Searched query = "Diamond-Blackfan 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.
| 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 | 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 |