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

February 1, 2024 updated by: Adrenas Therapeutics Inc

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

25

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Benioff Children's Hospital, Oakland
        • Sub-Investigator:
          • Tariq E Ahmad, MD, FAAP
        • Contact:
        • Principal Investigator:
          • Paul Harmatz, MD
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann and Robert H. Lurie Children's Hospital of Chicago
        • Contact:
        • Contact:
          • Phone Number: 312-227-6090
        • Principal Investigator:
          • Courtney Finlayson, MD
    • Maryland
      • Bethesda, Maryland, United States, 20892-1932
        • Recruiting
        • National Institutes of Health Clinical Center
        • Contact:
          • Office of Patient Recruitment
          • Phone Number: 800-411-1222
          • Email: prpl@cc.nih.gov
        • Contact:
          • Elizabeth Joyal (Study Coordinator), CRNP
          • Phone Number: 301-496-8542
          • Email: ejoyal@nih.gov
        • Principal Investigator:
          • Deborah Merke, M.D., M.S.
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
        • Contact:
        • Contact:
          • Coordinator Office
          • Phone Number: 612-626-7024
        • Principal Investigator:
          • Kyriakie Sarafoglou, M.D.
    • North Carolina
      • Morehead City, North Carolina, United States, 28557
        • Recruiting
        • Lucas Research, Inc.
        • Contact:
        • Principal Investigator:
          • K. Jean Lucas, MD,FACP,FACE
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Not yet recruiting
        • Children's Hospital of Philadelphia
        • Principal Investigator:
          • Maria Vogiatzi, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dose Level 1
BBP-631 lowest dose, administered once, intravenously (IV)
Biological: AAV BBP-631
intravenous
Experimental: Dose Level 2
BBP-631 middle dose, administered once, IV
Biological: AAV BBP-631
intravenous
Experimental: Dose Level 3
BBP-631, high dose, administered once, IV
Biological: AAV BBP-631
intravenous
Experimental: Dose Level 4
BBP-631, highest dose, administered once, IV
Biological: AAV BBP-631
intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Time Frame: up to 3 years
up to 3 years
To select the optimum dose or dose range of BBP 631 for future studies
Time Frame: up to 3 years
up to 3 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline in endogenous cortisol levels
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline in androstenedione (A4) levels
Time Frame: Baseline, Week 52
Baseline, Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Adrenas Therapeutics Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2021

Primary Completion (Estimated)

June 1, 2024

Study Completion (Estimated)

November 1, 2028

Study Registration Dates

First Submitted

March 1, 2021

First Submitted That Met QC Criteria

March 3, 2021

First Posted (Actual)

March 5, 2021

Study Record Updates

Last Update Posted (Actual)

February 2, 2024

Last Update Submitted That Met QC Criteria

February 1, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAH-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Congenital Adrenal Hyperplasia

Clinical Trials on AAV BBP-631

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ghana

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe