284. ダイアモンド・ブラックファン貧血 Diamond-Blackfan anemia Clinical trials / Disease details


臨床試験数 : 36 薬物数 : 95 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

  
2 trials found
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1NCT02386267
(ClinicalTrials.gov)
September 201418/2/2015L-leucine in Diamond Blackfan Anemia PatientsTherapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan Anemia PatientsDiamond Blackfan AnemiaDrug: L-leucineFederal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry RogacheNULLRecruiting1 Year20 YearsBoth30Phase 2Russian Federation
2NCT01362595
(ClinicalTrials.gov)
June 201320/5/2011Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan AnemiaThe Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance Using Bone Marrow Failure as a Model: a Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan AnemiaDiamond Blackfan Anemia;Blackfan Diamond Syndrome;DBA;Congenital Hypoplastic Anemia;Pure Red Cell AplasiaDrug: leucineNorthwell HealthNULLActive, not recruiting2 YearsN/AAll50Phase 1/Phase 2United States