NCT00125788已完成2 期
A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 5
- 主要终点
- Number of Occurrences of Painful Sickle Cell Crises
研究概览
简要总结
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
详细描述
The primary purpose of this study is to evaluate the effectiveness of oral L-glutamine in the therapy of sickle cell anemia and sickle ß0-thalassemia.
The secondary purpose is to assess the effect of L-glutamine frequency of hospitalizations for sickle cell pain, frequency of emergency room visits for sickle cell pain; energy and appetite levels; narcotics usage.
Methodology:
By site, patients will be randomized to L-glutamine or placebo in a 1:1 ratio after a 4-week screening period. Patients will undergo 48 weeks of treatment with dosing BID orally, with dose calculated according to patient weight. Patient visits will occur every 4 weeks. After 48 weeks of treatment, dose will be tapered to zero within 3 weeks. A final evaluation visit will occur 2 weeks after last dose.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 —（Child, 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 five 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.
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
investigational product
Experimental
L-glutamine
干预措施: L-glutamine (Drug)
placebo
Placebo Comparator
maltodextrin
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Occurrences of Painful Sickle Cell Crises
时间窗: From Week 0 through Week 48 (cumulative)
The mean number of painful sickle crisis through week 48
次要结局
- Frequency of Hospitalizations for Sickle Cell Pain(From Week 0 through Week 48 (cumulative))
- Frequency of Emergency Room Visits for Sickle Cell Pain(From Week 0 through Week 48 (cumulative))
- Number of Participants With Narcotic Usage(Week 24, Week 48)
- The Effect of Oral L-glutamine on Vital Signs - Blood Pressure(Baseline, Weeks 4, 24, and 48)
- Effect of Oral L--glutamine on Height(Baseline, Weeks 4, 24, and 48)
- Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest(Baseline, Weeks 4, 24, and 48.)
- Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest(Baseline, Weeks 4, 24, and 48.)
- The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin(Baseline, Weeks 4, 24 and 40)
- The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit(Baseline, Weeks 4, 24, and 40)
- The Effect of Oral L-glutamine on Vital Signs - Pulse Rate(Baseline, Weeks 4, 24, and 48)
- Effect of L-glutamine on Alcohol Use(Weeks 0, 8,16, 24, 32, 40 and 48)
- The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain(Weeks 0, 8,16, 24, 32, 40 and 48)
- Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest(Baseline, Weeks 4, 24, and 48.)
- Patient Appetite (3-point Scale)(Collected at Week 0, 8, 16, 24, 32, 40, 48)
- Effect of L-glutamine on Tobacco Use(Weeks 0, 8,16, 24, 32, 40 and 48)
- The Effect of Oral L-glutamine on Vital Signs - Respiration(Baseline, Weeks 4, 24, and 48)
- The Effect of Oral L-glutamine on Subjective Quality of Life(Baseline and Week 24 (or at time of discontinuation))
- Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest(Baseline, Weeks 4, 24, and 48.)
- The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count(Baseline, Weeks 0, 4, 24, 40)
- Energy Level (11-point Scale)(Collected at Week 0, 8, 16, 24, 32, 40, 48)
- The Effect of Oral L-glutamine on Vital Signs - Temperature(Baseline, Weeks 4, 24, and 48)
- Effect of Oral L--glutamine on Weight(Baseline, Weeks 4, 24 and 48)
研究者
研究点 (5)
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