A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Plain-language summary

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.

Trial details

Phase

N/A

Status

Completed

Sponsor

Adrenas Therapeutics Inc

Intervention

Pre-Screening

Start date

7/1/2021

Estimated completion

8/7/2024

Countries

United States

Related genes

Locations

• Los Angeles, United States

Eligibility criteria

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