Status:
ACTIVE_NOT_RECRUITING
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Lead Sponsor:
Adrenas Therapeutics Inc
Conditions:
Congenital Adrenal Hyperplasia
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
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.
Detailed Description
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.
Eligibility Criteria
Inclusion
- Key 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 and \< 40 × ULN (upper limit of normal)
- Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
- Naïve to prior gene therapy or AAV-mediated therapy
- Key Exclusion Criteria
- Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
- History of adrenalectomy and/or significant liver disease
Exclusion
Key Trial Info
Start Date :
July 1 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 1 2029
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT04783181
Start Date
July 1 2021
End Date
February 1 2029
Last Update
December 23 2025
Active Locations (5)
Enter a location and click search to find clinical trials sorted by distance.
1
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
2
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892-1932
3
University of Minnesota
Minneapolis, Minnesota, United States, 55455
4
Lucas Research, Inc.
Morehead City, North Carolina, United States, 28557