NCT04783181

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Adrenas Therapeutics Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Congenital Adrenal Hyperplasia

Interventions

Type	Name	Description
BIOLOGICAL	AAV BBP-631	intravenous

Timeline

• Start date: 2021-07-01
• Primary completion: 2029-02-01
• Completion: 2029-02-01
• First posted: 2021-03-05
• Last updated: 2025-12-23

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study