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

Adrenas Therapeutics

Status: Completed

Conditions: Congenital Adrenal Hyperplasia

Treatments: Other: Pre-Screening

Study type: Observational

Funder types: Industry

Identifiers

NCT05101902

CAH-300

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 65 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

65 participants in 1 patient group

Overall Study

Description:

Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility

Treatment:

Other: Pre-Screening