Clinical trial NCT04783181: A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Congenital Adrenal Hyperplasia
Keywords: CAH
Gene therapy
AAV
AAV5
Lead sponsor: Adrenas Therapeutics Inc
Sponsor class: INDUSTRY
First posted: 2021-03-05
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Participants will be assigned sequentially to one of 4 dose levels depending on the date of determination of eligibility.
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
timeFrame: up to 5 years
measure: To select the optimum dose or dose range of BBP 631 for future studies
timeFrame: up to 5 years
Secondary outcomes:
measure: Change from Baseline in 17-OHP (hydroxyprogesterone) levels
timeFrame: Baseline, Week 52 and through study completion, an average of 5 years
measure: Change from Baseline in androstenedione (A4) levels
timeFrame: Baseline, Week 52 and through study completion, an average of 5 years
measure: Change from Baseline in endogenous cortisol levels
timeFrame: Baseline, Week 52 and through study completion, an average of 5 years
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Morehead City
country: United States
facility: Lucas Research, Inc.
state: North Carolina
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
References: