The 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 Anemia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 55
- 试验地点
- 11
- 主要终点
- Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia
研究概览
简要总结
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.
The leucine is expected to produce a response in patients with DBA to the point where red blood cell production is increased. Red cell transfusions can then be less frequent or possibly discontinued.
The investigators will study the side effects, if any, of giving leucine to DBA patients. Leucine levels of leucine will be obtained at baseline and during the study.
The drug leucine will be provided in capsule form and taken 3 times a day for a total of 9 months.
详细描述
Leucine will be provided to participants in the form of a capsule and will be taken three times daily.
Blood hemoglobin levels will be monitored every 3-4 weeks for 9 months.
The entire study will last 12-15 months in length.
Subjects must be two years of age or older and on transfusion for more than six months prior to enrollment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed with Diamond Blackfan anemia as published in British Journal of Hematology
- •transfusion dependent
- •age 2 years and older
- •adequate renal function
- •adequate liver function
- •negative B-HCG if patient is a menstruating female and documentation of adequate contraception
- •signed informed consent
排除标准
- •Known hypersensitivity to branched chain amino acids
- •Diagnosis of an inborn error of amino acid metabolism disorder
- •Prior hematopoietic stem cell transplantation
- •Pregnancy, or plans to become pregnant during duration of trial
研究组 & 干预措施
Leucine
No alternative treatment arm
干预措施: leucine (Drug)
结局指标
主要结局
Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia
时间窗: 9 Months
The primary outcome is the type of response observed at 9 months. Response to treatment can be one of the following: 1. Complete response (CR): Hb \> 9 gm/dL and transfusion-independence as defined in DBA 2. Partial response (PR): Hb \< 9 gm/dL and increased reticulocyte count to greater than baseline. 3. No response (NR): no change in transfusion requirements and no significant change reticulocyte count from baseline 4. Progression: worsening of disease as defined by the need for more frequent transfusions
次要结局
- Severe Adverse Events Attributable to Leucine(9 months)
研究者
Adrianna Vlachos, MD
MD, Principal Investigator
Northwell Health