A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Phase 1
Recruiting
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Biological: AAV BBP-631
- Registration Number
- NCT04783181
- Lead Sponsor
- Adrenas Therapeutics Inc
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 25
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Dose Level 1 AAV BBP-631 BBP-631 lowest dose, administered once, intravenously (IV) Dose Level 4 AAV BBP-631 BBP-631, highest dose, administered once, IV Dose Level 2 AAV BBP-631 BBP-631 middle dose, administered once, IV Dose Level 3 AAV BBP-631 BBP-631, high dose, administered once, IV
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Change from Baseline in 17-OHP (hydroxyprogesterone) levels Baseline, Week 52 and through study completion, an average of 5 years Change from Baseline in androstenedione (A4) levels Baseline, Week 52 and through study completion, an average of 5 years Change from Baseline in endogenous cortisol levels Baseline, Week 52 and through study completion, an average of 5 years
Trial Locations
- Locations (5)
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
National Institutes of Health Clinical Center
🇺🇸Bethesda, Maryland, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Lucas Research, Inc.
🇺🇸Morehead City, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Ann and Robert H. Lurie Children's Hospital of Chicago🇺🇸Chicago, Illinois, United States