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

Sponsor

Adrenas Therapeutics Inc (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04783181

Collaborator

(none)

Enrollment

Locations

Arms

Anticipated Duration (Months)

3.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Congenital Adrenal Hyperplasia	Biological: AAV BBP-631	Phase 1/Phase 2

Detailed Description

Participants will receive a single dose of intravenous (IV) BBP-631 and are monitored for 52 weeks post-treatment. All participants who receive AAV5 based BBP-631 will be followed for an additional 4 years for safety and efficacy in a separate long-term follow-up study. In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

25 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Intervention Model Description:

Participants will be assigned sequentially to one of 3 dose levels depending on the date of determination of eligibility.Participants will be assigned sequentially to one of 3 dose levels depending on the date of determination of eligibility.

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jul 1, 2021

Anticipated Primary Completion Date :

Jun 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Dose Level 1 BBP-631 lowest dose, administered once, intravenously (IV)	Biological: AAV BBP-631 intravenous
Experimental: Dose Level 2 BBP-631 middle dose, administered once, IV	Biological: AAV BBP-631 intravenous
Experimental: Dose Level 3 BBP-631, highest dose, administered once, IV	Biological: AAV BBP-631 intravenous

Arm

Intervention/Treatment

Experimental: Dose Level 1

BBP-631 lowest dose, administered once, intravenously (IV)

Biological: AAV BBP-631

intravenous

Experimental: Dose Level 2

BBP-631 middle dose, administered once, IV

Biological: AAV BBP-631

intravenous

Experimental: Dose Level 3

BBP-631, highest dose, administered once, IV

Biological: AAV BBP-631

intravenous

Outcome Measures

Primary Outcome Measures

Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation [up to 3 years]
To select the optimum dose or dose range of BBP 631 for future studies [up to 3 years]

Secondary Outcome Measures

Change from Baseline in 17-OHP (hydroxyprogesterone) levels [Baseline, Week 52]
Change from Baseline in endogenous cortisol levels [Baseline, Week 52]
Change from Baseline in androstenedione (A4) levels [Baseline, Week 52]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria

Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
Screening/baseline 17-OHP levels > 5-10 × ULN and < 40 × ULN (upper limit of normal)
Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
Naïve to prior gene therapy or AAV-mediated therapy

Key Exclusion Criteria

Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
History of adrenalectomy and has no significant liver disease

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Los Angeles	Los Angeles	California	United States	90027
2	Rady Children's Hospital - San Diego	San Diego	California	United States	92123
3	Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	United States	60611
4	National Institutes of Health Clinical Center	Bethesda	Maryland	United States	20892-1932
5	University of Minnesota	Minneapolis	Minnesota	United States	55455
6	Lucas Research, Inc.	Morehead City	North Carolina	United States	28557
7	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104