A Phase 1/2a, Two-Part, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability and Pharmacodynamics of Multiple Doses of Oral BBI-001 in Healthy Participants and in Participants With Hereditary Hemochromatosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Adverse Events (AE)
研究概览
简要总结
This study will be conducted in two parts run in parallel.
Part 1 of the study (Phase 1, randomized, double-blind, placebo-controlled, multiple ascending dose) will enroll healthy volunteers in 3 sequential dose escalating cohorts with BBI-101 or placebo administered 3 times per day (TID) for 14 days.
Part 2 of the study (Phase 2a, randomized, multiple dose, two-period, two-sequence crossover) will evaluate the effect of BBI-001 on blood iron parameters in patients with hereditary hemochromatosis receiving 8 doses of BBI-001 or placebo treatment administered TID in Period 1 followed by administration of reverse treatment in Period 2. Dosing periods will be separated by 12 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Healthy volunteers (Part 1) or patients with hereditary hemochromatosis (Part 2)
排除标准
- •Serious or unstable medical or psychiatric conditions
- •Significant medical history
- •Current infections
- •Alcohol use disorder
- •Receiving iron chelation therapy or treatment other than stable maintenance phlebotomy for the prior 6 months (Part 2)
- •Organ damage from iron overload in the view of the principal investigator would prevent successful completion of the protocol (Part 2)
研究组 & 干预措施
Placebo
干预措施: Placebo (Other)
BBI-001
干预措施: BBI-001 (Drug)
结局指标
主要结局
Adverse Events (AE)
时间窗: Up to 30 days
Incidence and severity of AEs and serious AEs (SAEs), including clinically significant abnormalities in physical examinations, vitals signs, ECG parameters, and laboratory results
次要结局
未报告次要终点