A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
研究概览
简要总结
The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients
The secondary purpose is to assess the effect of L-glutamine on incidence of painful crises; level of chronic pain, and amount of daily requirement for narcotics.
详细描述
This is a phase II, prospective, randomized, double-blind, placebo-controlled, parallel-group,study to evaluate the safety and efficacy of oral L-glutamine therapy for patients with sickle cell anemia or sickle β°-thalassemia who are at least 18 years old with focus on the aspect of exercise endurance. In this study, the patients will orally take L-glutamine or placebo twice daily, and clinical and hematological parameters will be monitored. The adverse events,especially those attributable to L-glutamine therapy, will also be monitored.
The data obtained will be essential in understanding the role of L-glutamine in therapy for sickle cell anemia and sickle β°-thalassemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •To be eligible to participate in the study, a patient must meet all of the following inclusion criteria:
- •Patient is at least 18 years of age.
- •Patient has been diagnosed with sickle cell anemia or sickle ß0-thalassemia (documented by hemoglobin electrophoresis).
- •Patient has had at least two episodes of painful crises within 12 months of the screening visit.
- •If the patient has been treated with an anti-sickling agent within three months of the screening visit, the therapy must have been continuous for at least three months with the intent to continue for the next 14 months.
- •Patient or the patient's legally authorized representative has given written informed consent.
- •If the patient is a female of child-bearing potential, she agrees to practice a recognized form of birth control during the course of the study.
- •Patient is able to perform exercise tolerance test
排除标准
- •If the patient meets any of the following criteria, the patient must not be enrolled:
- •Patient has a significant medical condition that required hospitalization (other than sickle painful crisis) within two months of the screening visit.
- •Patient has diabetes mellitus with untreated fasting blood sugar >115 mg/dL.
- •Patient has prothrombin time International Normalized Ratio (INR) > 2.
- •Patient has serum albumin < 3.0 g/dl.
- •Patient has received any blood products within three weeks of the screening visit.
- •Patient has a history of uncontrolled liver disease or renal insufficiency.
- •Patient is pregnant or lactating.
- •Patient has been treated with an experimental anti-sickling medication/treatment (except hydroxyurea) within 30 days of the screening visit.
- •Patient has been treated with an experimental drug within 30 days of the screening visit.
- •There are factors that would, in the judgment of the investigator, make it difficult for the patient to comply with the requirements of the study.
研究组 & 干预措施
L-glutamine
L-glutamine group will be given at the following dosage:
17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily >66.7 at 15 g 2X daily
干预措施: L-Glutamine (Drug)
Placebo
Maltodextrin group will be given at the following dosage:
17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily >66.7 at 15 g 2X daily
干预措施: Placebo (Drug)
结局指标
主要结局
Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
时间窗: Baseline, Weeks 8 and 12
Exercise endurance will be examined at each visit. Change from baseline will be reported at weeks 8 and 12
Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients
时间窗: Baseline, Weeks 18 and 12
Breath by breath exercise will be examined at each visit. Change from baseline will be reported at weeks 8 and 12
次要结局
- Effect of Oral L-glutamine on Incidence of Painful Crises(From Week 0 through Week 12)
- Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics(From Week 0 through Week 20)
- Effect of Oral L-glutamine on Level of Chronic Pain(From Week 0 to Week 12)
研究者
yutaka niihara
President, Emmaus Medical
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center