Gene Therapy for Congenital Adrenal Hyperplasia
Trial Summary
Will I have to stop taking my current medications?
The trial requires participants to be on a stable oral hydrocortisone regimen as their only glucocorticoid maintenance therapy. If you are taking other medications, you may need to adjust your treatment to meet this requirement.
What data supports the effectiveness of the treatment AAV BBP-631 for congenital adrenal hyperplasia?
Is gene therapy using AAV BBP-631 generally safe in humans?
Research on gene therapy using adeno-associated virus (AAV) vectors, like AAV BBP-631, has shown that it can temporarily restore normal function in mice with congenital adrenal hyperplasia without causing an immune response. However, the effects were short-lived, and more research is needed to ensure long-term safety and effectiveness in humans.12367
How is the gene therapy AAV BBP-631 different from other treatments for congenital adrenal hyperplasia?
AAV BBP-631 is a gene therapy that uses a virus to deliver a functional copy of the mutated gene directly to the adrenal gland, aiming to restore normal hormone production. Unlike lifelong oral steroid treatments, this approach targets the root genetic cause of the condition, although its effects may be temporary due to the renewal of adrenal cells.12789
What is the purpose of this trial?
This trial tests a new gene therapy called BBP-631, delivered by a virus through an IV, in adults with classic congenital adrenal hyperplasia. The goal is to see if it is safe and effective over several years. BBP-631 is a gene therapy delivered by a virus, similar to other gene therapies that have shown promise in treating adrenal disorders.
Eligibility Criteria
Adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency, who haven't had gene therapy before and are on a stable dose of hydrocortisone. Pregnant women, those with previous adrenalectomy or significant liver disease, or immunity against AAV5 cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single dose of AAV5 based intravenous (IV) BBP-631
Follow-up
Participants are monitored for safety and efficacy for at least 5 years after treatment
Treatment Details
Interventions
- AAV BBP-631
Find a Clinic Near You
Who Is Running the Clinical Trial?
Adrenas Therapeutics Inc
Lead Sponsor