NCT05101902RecruitingNot Applicable
Pre-screening Study to Identify Adult Participants With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial With BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Adrenas Therapeutics Inc
- Enrollment
- 300
- Locations
- 1
- Primary Endpoint
- Number of Participants Potentially Eligible for CAH-301 Study
Study Overview
Brief Summary
This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.
Detailed Description
Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
- •Is on a daily regimen of glucocorticoid
- •Naïve to prior gene therapy or AAV-mediated therapy
Exclusion Criteria
- •Positive for anti-AAV5 antibodies
- •History of adrenalectomy and has no significant liver disease
Outcomes
Primary Outcomes
Number of Participants Potentially Eligible for CAH-301 Study
Time Frame: Up to 5 years
Secondary Outcomes
No secondary outcomes reported
Investigators
Study Sites (1)
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