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

NCT05101902 | Completed DMC FDA Drug

• 1 other identifier: CAH-300
• Study Type: observational
• Target: 65
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Congenital Adrenal Hyperplasia

Keywords

CAH

Outcome Measures

Primary Outcomes (1)

• Number of Participants Potentially Eligible for CAH-301 Study

  Up to 5 years

Study Arms (1)

Overall Study

Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility

Other: Pre-Screening

Interventions

Pre-Screening OTHER

No intervention given

Overall Study

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult participants diagnosed with classic CAH due to 21-OHD

You may qualify if:

• 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

You may not qualify if:

• Positive for anti-AAV5 antibodies
• History of adrenalectomy and has no significant liver disease

Sponsors & Collaborators

• Adrenas Therapeutics Inc: lead

Study Sites (1)

Science37

Los Angeles, California, 90094, United States

Location

Related Links

• Description: Adrenas Therapeutics Website
• CAH Gene Therapy Website

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2021
• First Posted: November 1, 2021
• Study Start: July 1, 2021
• Primary Completion: August 7, 2024
• Study Completion: August 7, 2024
• Last Updated: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will share

Locations

US (1)