NCT05101902

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

Pre-screening Study to Identify Adult Participants with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial with BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 65 (actual)
• Sponsor: Adrenas Therapeutics Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Congenital Adrenal Hyperplasia

Interventions

Type	Name	Description
OTHER	Pre-Screening	No intervention given

Timeline

• Start date: 2021-07-01
• Primary completion: 2024-08-07
• Completion: 2024-08-07
• First posted: 2021-11-01
• Last updated: 2024-09-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study