L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

L-谷氨酰胺治疗镰状细胞性贫血

• 批准号: 7606151
• 负责人: RICHARD CASABURI
• 金额: $ 0.72万
• 依托单位: LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-02-01 至 2007-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7606151
• 关键词: 18 year old; Affect; African; Biochemical; Clinical; Clinical Data; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Data; Diet; Double-Blind Method; End Point; Erythrocytes; Evaluable Disease; Exercise; Funding; Glutamine; Goals; Grant; Hereditary Disease; Individual; Institution; International; Modality; Morbidity - disease rate; Oral; Orphan Drugs; Patients; Perception; Phase; Physiological; Pilot Projects; Randomized; Research; Research Personnel; Resources; Safety; Sickle Cell Anemia; Source; Testing; Therapeutic; United States Food and Drug Administration; United States National Institutes of Health; chronic pain; dosage; mortality; pulmonary function

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Sickle cell anemia is a hereditary disorder with significant morbidity and mortality affecting mostly individuals of African heritage. Currently approved therapeutic modalities have their limitations in safety, efficacy and availability. Recent data demonstrate potential for L-glutamine as an oral agent for therapy of sickle cell disease. Pilot studies have brought forth promising results in physiological parameters. Also notable was the consistent improvements in patients' subjective perception of clinical status especially in their energy levels and chronic pain. The goal of Orphan Drugs International is to evaluate application of L-glutamine therapy in sickle cell disease for potential FDA approval. With this proposal, a randomized double blind clinical trial with objective clinical endpoints is planned. This will test the claims of subjective clinical data with objective endpoints. During the phase I period of the project, 20 evaluable sickle cell anemia patients (18 years and older) will be studied. The primary endpoint will be exercise endurance with pulmonary function of the patients. The secondary endpoints will be physiologic and biochemical parameters of red blood cells. L-glutamine is very much a part of our regular diet and there are ample data on its safety at the dosage used in this project.

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 镰状细胞性贫血是一种遗传性疾病，发病率和死亡率很高，主要影响非洲血统的人。目前批准的治疗方式在安全性、有效性和可用性方面存在局限性。最近的数据表明，L-谷氨酰胺作为一种口服药物治疗镰状细胞病具有潜力。初步研究在生理参数方面取得了令人振奋的结果。同样值得注意的是，患者对临床状态的主观感觉持续改善，特别是在他们的能量水平和慢性疼痛方面。国际孤儿药物协会的目标是评估L-谷氨酰胺疗法在镰状细胞疾病中的应用，以获得美国食品和药物管理局的批准。根据这一建议，计划进行一项具有客观临床终点的随机双盲临床试验。这将用客观的终点来检验主观临床数据的说法。在该项目的第一阶段期间，将对20名可评估的镰状细胞性贫血患者(18岁及以上)进行研究。主要终点是运动耐力和患者的肺功能。次级终点将是红细胞的生理和生化参数。L-谷氨酰胺在很大程度上是我们日常饮食的一部分，在本项目使用的剂量下，有大量关于其安全性的数据。