A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
Recruiting
- Conditions
- Congenital Adrenal Hyperplasia
- Registration Number
- NCT05101902
- Lead Sponsor
- Adrenas Therapeutics Inc
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 300
Inclusion Criteria
- 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
Exclusion Criteria
- Positive for anti-AAV5 antibodies
- History of adrenalectomy and has no significant liver disease
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants Potentially Eligible for CAH-301 Study Up to 5 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Science37
🇺🇸Los Angeles, California, United States
Science37🇺🇸Los Angeles, California, United States