A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Sponsored by Adrenas Therapeutics Inc
About this trial
Last updated 10 months ago
Study ID
CAH-301
Status
Active, not recruiting
Type
Interventional
Phase
Phase 1/Phase 2
Placebo
No
Accepting
18+ Years
All
Not accepting
Healthy Volunteers
Trial Timing
Started 4 years ago
What is this trial about?
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.
What are the Participation Requirements?
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