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

a study on Congenital Adrenal Hyperplasia

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Paul Harmatz, MD

Description

Summary

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.

Official Title

A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

Details

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.

Keywords

Congenital Adrenal Hyperplasia, CAH, Gene therapy, AAV, AAV5, Adrenogenital Syndrome, Adrenocortical Hyperfunction, Hyperplasia, AAV BBP-631

Eligibility

You can join if…

Open to people ages 18 years and up

  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 CAN'T join if...

  1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
  2. History of adrenalectomy and/or significant liver disease

Locations

  • UCSF Benioff Children's Hospital, Oakland accepting new patients
    Oakland California 94609 United States
  • University of Minnesota accepting new patients
    Minneapolis Minnesota 55455 United States

Lead Scientist at UCSF

  • Paul Harmatz, MD
    Professor, Pediatrics, School of Medicine. Authored (or co-authored) 213 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Adrenas Therapeutics Inc
Links
Adrenas Therapeutics Website CAH Gene Therapy Website
ID
NCT04783181
Phase
Phase 1/2 Congenital Adrenal Hyperplasia Research Study
Study Type
Interventional
Participants
Expecting 25 study participants
Last Updated
Contact us about this trial