Gene Therapy for Congenital Adrenal Hyperplasia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy treatment called AAV BBP-631 for individuals with classic congenital adrenal hyperplasia (CAH), a condition that prevents the body from producing certain essential hormones. The study aims to determine the treatment's safety, tolerance, and effectiveness in managing CAH symptoms. Participants will receive one of four different doses of the treatment through an IV. Suitable candidates have classic CAH, use oral hydrocortisone as their only maintenance therapy, and have not undergone previous gene therapy. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.
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.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that the gene therapy BBP-631 was well tolerated in studies. Participants experienced only mild to moderate side effects, indicating no serious health issues arose from the treatment. No severe side effects were directly linked to it. This evidence suggests that BBP-631 is relatively safe. However, the development of this treatment has been stopped, which might affect its availability or further testing.12345
Why do researchers think this study treatment might be promising for congenital adrenal hyperplasia?
Unlike the standard of care for congenital adrenal hyperplasia, which often involves lifelong hormone replacement therapy, BBP-631 offers a gene therapy approach using an AAV vector. This treatment is unique because it targets the root cause of the condition by delivering a functional gene to correct the enzyme deficiency. Researchers are excited about BBP-631 because it has the potential to provide a one-time treatment that could significantly reduce or eliminate the need for ongoing medication, offering a more sustainable and long-term solution.
What evidence suggests that AAV BBP-631 might be an effective treatment for congenital adrenal hyperplasia?
Research has shown that the gene therapy BBP-631 offers promising results for treating classic congenital adrenal hyperplasia (CAH). In this trial, participants will receive varying doses of BBP-631, from the lowest to the highest. Studies found that patients who received higher doses experienced an increase in their body's natural cortisol production. Cortisol, a hormone, aids in stress management and metabolism. This increase marked a first-of-its-kind result for these patients. Despite these positive changes, the company chose not to continue developing the therapy because the overall results were not strong enough.12346
Are You a Good Fit for This Trial?
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 for a Trial Participant
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
What Are the Treatments Tested in This Trial?
Interventions
- AAV BBP-631
Find a Clinic Near You
Who Is Running the Clinical Trial?
Adrenas Therapeutics Inc
Lead Sponsor