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NCT04783181 Clinical Trial

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

Congenital Adrenal Hyperplasia Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT04783181
Other study ID # CAH-301
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date July 1, 2021
Est. completion date November 2028

Study information
Verified date February 2024
Source Adrenas Therapeutics Inc
Contact Patient Advocacy
Phone 650-391-9740
Email ClinicalTrials@AdrenasTx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 25
Est. completion date November 2028
Est. primary completion date June 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Related Conditions & MeSH terms

Intervention

Biological:
• AAV BBP-631
intravenous

Locations
Country Name City State
United States National Institutes of Health Clinical Center Bethesda Maryland
United States Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States University of Minnesota Minneapolis Minnesota
United States Lucas Research, Inc. Morehead City North Carolina
United States UCSF Benioff Children's Hospital, Oakland Oakland California
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania

Sponsors (1)
Lead Sponsor Collaborator
Adrenas Therapeutics Inc

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation up to 3 years
Primary To select the optimum dose or dose range of BBP 631 for future studies up to 3 years
Secondary Change from Baseline in 17-OHP (hydroxyprogesterone) levels Baseline, Week 52
Secondary Change from Baseline in endogenous cortisol levels Baseline, Week 52
Secondary Change from Baseline in androstenedione (A4) levels Baseline, Week 52
See also
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Completed NCT02934399 - Dynamic Hormone Diagnostics in Endocrine Disease
Recruiting NCT04903587 - Gonadal Changes In Congenital Adrenal Hyperplasia Patients
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Active, not recruiting NCT04490915 - Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia Phase 3
Recruiting NCT05663320 - A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia N/A
Completed NCT01807364 - Cardiovascular Risk Profile in Patients With Congenital Adrenal Hyperplasia N/A
Completed NCT02804178 - A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia Phase 2
Completed NCT03019614 - An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone Phase 1
Not yet recruiting NCT04293133 - Final Height in Patients With CAH
Recruiting NCT03897504 - Surgical Evaluation of Using the Prepuce in Feminizing Genitoplasty N/A
Active, not recruiting NCT04806451 - Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study) Phase 3

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