This is a pre-screening study to determine whether patients with classic congenital adrenal hyperplasia (CAH) are eligible to take part in the CAH-301 gene therapy trial. It assessed adults with classic CAH due to 21-OHD deficiency who take daily glucocorticoids and have not previously received gene therapy. This study has no assigned phase and has been completed.
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.
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