CLINICAL TRIAL OF ISOBUTYRAMIDE IN BETA THALASSEMIA AND SICKLE CELL ANEMIA

异丁酰胺治疗β地中海贫血和镰状细胞贫血症的临床试验

• 批准号: 3763686
• 负责人: DAN COSTIN
• 金额: --
• 依托单位: NEW YORK UNIVERSITY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3763686
• 关键词: amides; blood disorder chemotherapy; butyrates; clinical trials; dosage; drug screening /evaluation; gamma globulin; human subject; human therapy evaluation; sickle cell anemia; thalassemia

In this study, isobutyramide, a derivative of butyric acid, will be investigated to establish the maximally tolerated dose of isobutyramide and to determine if isobutyramide can stimulate gamma globin synthesis in patients with beta Thalassemia and sickle cell anemia.

在这项研究中，异丁酰胺，丁酸的衍生物，将 研究以确定异丁酰胺的最大耐受剂量 并确定异丁酰胺是否能刺激γ珠蛋白的合成 β地中海贫血和镰状细胞性贫血的患者。