A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) (NCT04783181) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
United States8 participantsStarted 2021-07-01
Plain-language summary
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 and \< 40 × ULN (upper limit of normal)
. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
. Naïve to prior gene therapy or AAV-mediated therapy
Exclusion criteria
. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
. History of adrenalectomy and/or significant liver disease
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Timeframe: up to 5 years
2
To select the optimum dose or dose range of BBP 631 for future studies