Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Adrenas Therapeutics Inc
Participants will receive a single dose of AAV5 based intravenous (IV) BBP-631 and will be followed for safety and efficacy for at least 5 years after the date of treatment with BBP-631.
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
2. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal)
3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
4. Naïve to prior gene therapy or AAV-mediated therapy
You may not be if
1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
2. History of adrenalectomy and/or significant liver disease
