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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria
1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
2. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal)
3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
4. Naïve to prior gene therapy or AAV-mediated therapy
Key Exclusion Criteria
1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
2. History of adrenalectomy and/or significant liver disease
Primary outcome measure(s)
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation — up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies — up to 5 years
Trial sites (5)
Facility
City
Region
Status
Ann and Robert H. Lurie Children's Hospital of Chicago
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.