A PHASE III, PROSPECTIVE, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTER STUDY OF L GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA AND SICKLE ß0-THALASSEMIA
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 230
- 试验地点
- 31
- 主要终点
- The Number of Occurrences of 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 or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
详细描述
Primary objective:
To evaluate the efficacy of oral L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia as evaluated by the number of occurrences of sickle cell crises.
Secondary objectives:
To assess the effect of oral L-glutamine on: (a) frequency of hospitalizations for sickle cell pain; (b) frequency of emergency room/medical facility visits for sickle cell pain; and (c) hematological parameters (hemoglobin, hematocrit, and reticulocyte count); and to assess the safety of L-glutamine as a therapy for sickle cell anemia as evaluated by adverse events, laboratory parameters, and vital signs.
Methodology:
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
- •Patient is at least five years of age.
- •Patient has been diagnosed with sickle cell anemia or sickle ß°-thalassemia (documented by hemoglobin electrophoresis).
- •Patient has had at least two documented episodes of sickle cell 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 duration of the study.
- •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 avoid pregnancy during the study and is willing and agrees to practice a recognized form of birth control during the course of the study (e.g. barrier, birth control pills, abstinence).
排除标准
- •Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit.
- •Patient has prothrombin time 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 uncontrolled liver disease or renal insufficiency.
- •Patient is pregnant or lactating or has the intention of becoming pregnant during the study (if female and of child-bearing potential).
- •Patient is currently taking or has been treated with any form of glutamine supplement within 30 days of the screening visit.
- •Patient has been treated with an experimental anti-sickling medication/ treatment within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients).
- •Patient is currently taking or has been treated with an investigational drug within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients).
- •Patient is currently enrolled in an investigational drug or device study and/or has participated in such a study 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
Patients will be randomized to receive investigational product, L-Glutamine.
干预措施: L-glutamine (Drug)
100% maltodextrin
Patients will be randomized to receive Placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
The Number of Occurrences of Sickle Cell Crises
时间窗: 48 weeks
The number of occurrences of protocol-defined sickle cell crises that occur from Week 0 to Week 48 will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
次要结局
- The Effect of Oral -L-glutamine on Hematological Parameters(Baseline, Week 4, 24 and 48)
- The Effect of Oral L-glutamine on Hematological Parameters(Baseline, Week 4, 24 and 48)
- The Effect of Oral L-glutamine on Vital Signs(Baseline, Week 4, Week 24 and Week 48)
- The Number of Hospitalizations for Sickle Cell Pain(48 weeks)
- The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain(48 weeks)
- Effect of Oral L-glutamine on Vital Signs(Baseline, Week 4, Week 24 and Week 48)