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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
8
intravenous
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
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Time frame: up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies
Time frame: up to 5 years
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in androstenedione (A4) levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in endogenous cortisol levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States