A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

  • End date
    Nov 19, 2028
  • participants needed
  • sponsor
    Adrenas Therapeutics Inc
Updated on 7 October 2022
oral hydrocortisone
classic congenital adrenal hyperplasia


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.


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.

Condition Congenital Adrenal Hyperplasia
Treatment AAV BBP-631
Clinical Study IdentifierNCT04783181
SponsorAdrenas Therapeutics Inc
Last Modified on7 October 2022


Yes No Not Sure

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 has no significant liver disease
Clear my responses

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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