A Phase I Open Label, Unblinded, Single Dose Study for Evaluating the Safety and Pharmacokinetics of SANGUINATE™ in Sickle Cell Disease (SCD) Patients
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Evaluate the safety and tolerability of two dose regimen for SANGUINATE™ in sickle cell disease patients
研究概览
简要总结
Prolong proposes to test safety, tolerability and pharmacokinetics of SANGUINATE™ in sickle cell disease (SCD) patients. Prolong's preclinical studies showed that SANGUINATE™ was safe in a number of different animal models and toxicology studies. In this Phase I trial, Prolong will test whether it is also safe and tolerable in sickle cell patients. The study will be conducted in 15 adult (>18 years) patients.
详细描述
This Phase I trial will test two doses of SANGUINATE™ in adult subjects suffering from chronic sickle cell disease (SCD). A total of fifteen (15) patients will be assigned to the low and high dose cohorts of SANGUINATE™.
The first 8 subjects will receive the study agent in an initial low dose of 160 mg/kg of SANGUINATE™.
Upon completion of the low dose cohort, safety variables will be reviewed by the Principal Investigator, the Study Manager, and the Sponsor (or designee) in order to determine the progression towards administration of a high dose cohort of 320 mg/kg of SANGUINATE™. 7 subjects will be enrolled in the high dose cohort. Subjects will not be enrolled in the next higher dose cohort until the dose in the preceding dose group is deemed safe and tolerable.
Sample Size: A total of 15 patients will be enrolled in the study (8 patients in the low dose cohort, 7 patients in the high dose cohort). In both cohorts an additional participant would not be enrolled prior to safety review of the results of the previously enrolled participant.
Dose and Mode of Administration: SANGUINATE™ is PEGylated bovine hemoglobin (PEG-Hb) in the CO form formulated in saline (PEG-Hb-CO).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Evaluate the safety and tolerability of two dose regimen for SANGUINATE™ in sickle cell disease patients
时间窗: 7 days
The following assessments will be used to evaluate the safety of SANGUINATE™ administration: * adverse events, serious adverse events * laboratory abnormalities by highest toxicity grade * laboratory abnormalities by largest increase in toxicity grade from baseline
次要结局
- Determine the plasma pharmacokinetic (PK) profile of SANGUINATE™ in sickle cell disease patients. Mean values by treatment received will be calculated for the following PK parameters for PEGylated bovine hemoglobin:(7 Days)