A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 25
- 试验地点
- 5
- 主要终点
- Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
研究概览
简要总结
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
详细描述
Participants will receive a single dose of AAV5 based intravenous (IV) BBP-631 and will be followed for safety and efficacy for at least 5 years after the date of treatment with BBP-631.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（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
研究组 & 干预措施
Dose Level 1
BBP-631 lowest dose, administered once, intravenously (IV)
干预措施: AAV BBP-631 (Biological)
Dose Level 2
BBP-631 middle dose, administered once, IV
干预措施: AAV BBP-631 (Biological)
Dose Level 3
BBP-631, high dose, administered once, IV
干预措施: AAV BBP-631 (Biological)
Dose Level 4
BBP-631, highest dose, administered once, IV
干预措施: AAV BBP-631 (Biological)
结局指标
主要结局
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
时间窗: up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies
时间窗: up to 5 years
次要结局
- Change from Baseline in 17-OHP (hydroxyprogesterone) levels(Baseline, Week 52 and through study completion, an average of 5 years)
- Change from Baseline in androstenedione (A4) levels(Baseline, Week 52 and through study completion, an average of 5 years)
- Change from Baseline in endogenous cortisol levels(Baseline, Week 52 and through study completion, an average of 5 years)