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.

Study Timeline

• Study First Submitted: 2021-03-01
• Study First Submitted QC: 2021-03-03
• Study First Posted: 2021-03-05
• Study Start: 2021-07-01
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-05
• Last Update Posted: 2024-08-06
• Primary Completion: 2024-12
• Study Completion: 2028-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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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