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NCT05101902 Clinical Trial

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Congenital Adrenal Hyperplasia Clinical Trial

Official title:
Pre-screening Study to Identify Adult Participants With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial With BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT05101902
Other study ID # CAH-300
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date July 1, 2021
Est. completion date June 2024

Study information
Verified date July 2023
Source Adrenas Therapeutics Inc
Contact Patient Advocacy
Phone 650-391-9740
Email ClinicalTrials@AdrenasTX.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.

Description:

Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.

Recruitment information / eligibility
Status Recruiting
Enrollment 300
Est. completion date June 2024
Est. primary completion date June 2024
Accepts healthy volunteers
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD - Screening/baseline 17-OHP levels > 5-10 × ULN - Is on a daily regimen of glucocorticoid - Naïve to prior gene therapy or AAV-mediated therapy Exclusion Criteria: - Positive for anti-AAV5 antibodies - History of adrenalectomy and has no significant liver disease

Study Design

Related Conditions & MeSH terms

Intervention

Other:
• Pre-Screening
No intervention given

Locations
Country Name City State
United States Science37 Los Angeles California

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 Potentially Eligible for CAH-301 Study Up to 3 years
See also
  Status Clinical Trial Phase
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Recruiting NCT04903587 - Gonadal Changes In Congenital Adrenal Hyperplasia Patients
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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
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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
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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