This study examines AAV BBP-631, a gene therapy, in adults with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. It enrolls adult men and non-pregnant women with confirmed disease who are on a stable oral hydrocortisone regimen and have not previously received gene therapy. The study is being conducted in Phase 1/Phase 2 and is active but not currently recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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