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

Adrenas Therapeutics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Congenital Adrenal Hyperplasia

Treatments: Biological: AAV BBP-631

Study type: Interventional

Funder types: Industry

Identifiers

NCT04783181

CAH-301

Details and patient eligibility

About

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.

Full 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.

Enrollment: 8 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria

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

Key Exclusion Criteria

1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
2. History of adrenalectomy and/or significant liver disease

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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8 participants in 4 patient groups

Dose Level 1

Experimental group

Description:

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

Treatment:

Biological: AAV BBP-631

Dose Level 2

Experimental group

Description:

BBP-631 middle dose, administered once, IV

Treatment:

Biological: AAV BBP-631

Dose Level 3

Experimental group

Description:

BBP-631, high dose, administered once, IV

Treatment:

Biological: AAV BBP-631

Dose Level 4

Experimental group

Description:

BBP-631, highest dose, administered once, IV

Treatment:

Biological: AAV BBP-631