Gene Therapy for Congenital Adrenal Hyperplasia

Recruiting in Palo Alto (17 mi)

+8 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Adrenas Therapeutics Inc

No Placebo Group

Trial Summary

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

I have never received gene therapy or AAV-mediated therapy.

I am on a stable dose of oral hydrocortisone as my only steroid treatment.

Your 17-OHP levels are too high or too low during the screening test.

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Exclusion Criteria

I have had surgery to remove my adrenal gland or have significant liver disease.

You have antibodies against the AAV5 virus.

Treatment Details

Interventions

AAV BBP-631 (Gene Therapy)

Trial OverviewThe trial is testing the safety and effectiveness of a gene therapy called AAV BBP-631 in adults with CAH. It aims to see if this treatment can manage the condition better than current therapies.

Participant Groups

4Treatment groups

Experimental Treatment

Group I: Dose Level 4Experimental Treatment1 Intervention

BBP-631, highest dose, administered once, IV

Group II: Dose Level 3Experimental Treatment1 Intervention

BBP-631, high dose, administered once, IV

Group III: Dose Level 2Experimental Treatment1 Intervention

BBP-631 middle dose, administered once, IV

Group IV: Dose Level 1Experimental Treatment1 Intervention

BBP-631 lowest dose, administered once, intravenously (IV)