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

Last updated: December 19, 2025
Sponsor: Adrenas Therapeutics Inc
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

Male Hormonal Deficiencies/abnormalities

Congenital Adrenal Hyperplasia

Treatment

AAV BBP-631

Clinical Study ID

NCT04783181
CAH-301
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria

  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

Key Exclusion Criteria

  1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies

  2. History of adrenalectomy and/or significant liver disease

Study Design

Total Participants: 8
Treatment Group(s): 1
Primary Treatment: AAV BBP-631
Phase: 1/2
Study Start date:
July 01, 2021
Estimated Completion Date:
February 28, 2029

Study Description

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.

Connect with a study center

  • PARC Clinical Research, CALHN Royal Adelaide Hospital

    Adelaide, South Australia 5000
    Australia

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Site Not Available

  • UCSF Benioff Children's Hospital, Oakland

    Oakland, California 94609
    United States

    Site Not Available

  • Rady Children's Hospital - San Diego

    San Diego, California 92123
    United States

    Site Not Available

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago 4887398, Illinois 4896861 60611
    United States

    Site Not Available

  • National Institutes of Health Clinical Center

    Bethesda, Maryland 20892-1932
    United States

    Site Not Available

  • National Institutes of Health Clinical Center

    Bethesda 4348599, Maryland 4361885 20892-1932
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis 5037649, Minnesota 5037779 55455
    United States

    Site Not Available

  • Lucas Research, Inc.

    Morehead City, North Carolina 28557
    United States

    Site Not Available

  • Lucas Research, Inc.

    Morehead City 4480153, North Carolina 4482348 28557
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Site Not Available

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