NCT02314689已完成1 期
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
Suvankar Majumdar1 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2015年1月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria
研究概览
简要总结
The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.
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
- 18 Years 至 25 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)
排除标准
- •Presence of any acute illness defined by fever >100.4° F within the past 48 hours
- •Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
- •Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.
- •Severe anemia (hemoglobin < 5g/dL)
- •History of red blood cell transfusion within the last 14 days
- •Systemic steroid therapy within the last 48 hours
- •Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
- •Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.
- •Elevated serum creatinine >1.5mg/dL
- •Patients with an inability to give consent will be excluded
- •Medications that are known to be contra-indicated with use of L-citrulline (concurrent use of hydroxyurea will be allowed).
- •History of diabetes due to risk of electrolyte imbalance
研究组 & 干预措施
IV citrulline
Experimental
IV citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg.
干预措施: Intravenous (IV) citrulline (Drug)
结局指标
主要结局
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria
时间窗: 12 months
次要结局
未报告次要终点
研究者
Suvankar Majumdar
Associate Professor
University of Mississippi Medical Center
研究点 (1)
Loading locations...
相似试验
已完成
1 期
A Dose Escalation Trial of an Intravitreal Injection of Sirna-027 in Patients With Subfoveal Choroidal Neovascularization (CNV) Secondary to Age-Related Macular Degeneration (AMD)Age-Related Macular DegenerationChoroidal NeovascularizationNCT00363714Allergan26
已完成
1 期
L-Citrulline Dose Finding Safety Study in MELASMELAS SyndromeNCT03952234Baylor College of Medicine10
已完成
1 期
Evaluation of the Safety and Tolerability of i.v. Administration of a Cancer Vaccine in Patients With Advanced MelanomaMelanomaNCT02410733BioNTech SE119
已完成
2 期
Intravitreal Microplasmin in Patients Undergoing Surgical VitrectomyVitreomacular Traction MaculopathyEye DiseasesNCT00123279ThromboGenics60
已完成
1 期
Citrulline for Children Undergoing Cardiopulmonary Bypass SurgeryCardiovascular DiseasesHeart DiseasesHeart Defects, CongenitalNCT00201214Vanderbilt University26