NCT05101902招募中不适用
Pre-screening Study to Identify Adult Participants With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial With BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- Number of Participants Potentially Eligible for CAH-301 Study
研究概览
简要总结
This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.
详细描述
Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
- •Screening/baseline 17-OHP levels > 5-10 × ULN
- •Is on a daily regimen of glucocorticoid
- •Naïve to prior gene therapy or AAV-mediated therapy
排除标准
- •Positive for anti-AAV5 antibodies
- •History of adrenalectomy and has no significant liver disease
结局指标
主要结局
Number of Participants Potentially Eligible for CAH-301 Study
时间窗: Up to 5 years
次要结局
未报告次要终点
研究者
研究点 (1)
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