clinical trial · NCT04783181
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Adrenas Therapeutics Inc·phase2·active not recruiting·n = 8
Congenital Adrenal HyperplasiaAAV BBP-631
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.
started
Jul 1, 2021
primary completion
Feb 1, 2029
completion
Feb 1, 2029
last updated
Dec 23, 2025
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.
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol