A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 79
- 试验地点
- 3
- 主要终点
- BBP-671 concentration dependent change in change from baseline in QTcF
研究概览
简要总结
The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
详细描述
This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for future clinical studies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 15 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
BBP-671 for SAD
The SAD portion of the study will consist of up to 8 cohorts. Six (6) healthy male or female adult subjects will be randomized to receive BBP-671 per cohort (6:2 ratio, BBP-671:placebo).
干预措施: BBP-671 (Drug)
Placebo for SAD
The SAD portion of the study will consist of up to 8 cohorts. Two (2) healthy male or female adult subjects will be randomized to receive matching placebo per cohort (6:2 ratio, BBP-671:placebo).
干预措施: Placebo (Drug)
BBP-671 for MAD
The MAD portion of the study will consist of up to 6 cohorts. Six (6) healthy male or female adult subjects will be randomized to receive BBP-671 per cohort (6:2 ratio, BBP-671:placebo).
干预措施: BBP-671 (Drug)
Placebo for MAD
The MAD portion of the study will consist of up to 6 cohorts. Two (2) healthy male or female adult subjects will be randomized to receive matching placebo per cohort (6:2 ratio, BBP-671:placebo).
干预措施: Placebo (Drug)
BBP-671 for SAD Food Effect
Eight (8) healthy male or female adult subjects will be randomized to receive BBP-671.
干预措施: BBP-671 (Drug)
BBP-671 for PA and MMA Patients
Up to sixteen (16) patients with either PA or MMA will receive BBP-671.
干预措施: BBP-671 (Drug)
结局指标
主要结局
BBP-671 concentration dependent change in change from baseline in QTcF
时间窗: 49 days
Incidence of adverse events following administration of BBP-671
时间窗: 49 days
Pharmacokinetic Assessments: Vz/F
时间窗: 15 days
Apparent volume of distribution (Vz/F)
Pharmacokinetic Assessments: Cmax
时间窗: 49 days
Time to maximum concentration (Cmax)
Pharmacokinetic Assessments: Tmax
时间窗: 49 days
Time to reach maximum observed plasma concentration (Tmax)
Pharmacokinetic Assessments: t1/2
时间窗: 49 days
Plasma decay half-life (t1/2)
Pharmacokinetic Assessments: CLr
时间窗: 15 days
Renal clearance (CLr)
Pharmacokinetic Assessments: AUC0-tau
时间窗: 49 days
Area under the plasma concentration-time curve (AUC0-tau)
Pharmacokinetic Assessments: CL/F
时间窗: 15 days
Apparent clearance (CL/F)
次要结局
- Food Effect: Tmax(10 days)
- Food Effect: Cmax(10 days)
- Food Effect: AUC(10 days)
- Pharmacodynamic Assessment: Whole blood, plasma, and urine biomarker concentrations will be quantified and summarized using appropriate descriptive parameters(49 days)