PHASE II TRIAL OF BUTYRATE IN THE BETA HEMOGLOBINOPATHIES

丁酸盐治疗 β 型血红蛋白病的 II 期试验

• 批准号: 6278411
• 负责人: SUSAN Park PERRINE
• 金额: $ 2.92万
• 依托单位: BOSTON UNIVERSITY MEDICAL CAMPUS
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-12-01 至 1998-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6278411
• 关键词: aminobutyrate; analog; blood disorder chemotherapy; butyrates; clinical research; clinical trials; disease /disorder prevention /control; gene expression; gene induction /repression; hemoglobin F; human subject; human therapy evaluation; immunotherapy; outcomes research; sickle cell anemia; thalassemia

Phase II trials are proposed now to confirm that low-doses,intermittent regiments of butyrate produce hematolic efficacy in larger sample sizes. There is no standard therapy for thalassemia. In some patients, continued expression of small amounts of fetal globin has completely prevented clinical disease in these disorders, suggesting that, continued expression of the adult fetal globin gene after birth will prevent manifestations of disease. Chemotherapeutic agents are toxic in children. The aim of this proposal is to apply a new class of compound, isobutyramide, an analogue of butyric acid, to reactivate the fetal globin gene expression.

现在建议进行II期试验，以确认低剂量，间歇性