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

N/ACompletedNCT05101902

Adrenas Therapeutics Inc65 enrolled

Overview

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.

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 Type

OBSERVATIONAL

Enrollment

Conditions

Congenital Adrenal Hyperplasia

Interventions

Pre-ScreeningOTHER

No intervention given

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Science37

Los Angeles, California, United States

Outcomes

Primary Outcomes

Number of Participants Potentially Eligible for CAH-301 Study

Time frame: Up to 5 years

Data from ClinicalTrials.gov

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