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

Completed

Conditions studied: Congenital Adrenal Hyperplasia

In brief

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.

Key facts

Study ID

NCT05101902

Run by

Adrenas Therapeutics Inc

People needed

65

Starts

2021-07-01

Expected to finish

2024-08-07

Last updated by the study team

2024-09-19

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Positive for anti-AAV5 antibodies
• History of adrenalectomy and has no significant liver disease

Where it is running

• Science37 — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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