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A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Sponsor: Adrenas Therapeutics Inc
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.
Official title: A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
8
Start Date
2021-07-01
Completion Date
2029-02
Last Updated
2025-12-23
Healthy Volunteers
No
Conditions
Interventions
AAV BBP-631
intravenous
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