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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05101902

Enrollment

Registered

2021-11-01

Start date

2021-07-01

Completion date

2024-08-07

Last updated

2024-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Adrenal Hyperplasia

Keywords

CAH

Brief 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.

Interventions

OTHERPre-Screening

No intervention given

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame
Number of Participants Potentially Eligible for CAH-301 Study	Up to 5 years

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 11, 2026