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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Science37

Los Angeles, California, 90094, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Pre-Screening

Other

No intervention given

Primary outcomes

Number of Participants Potentially Eligible for CAH-301 Study

Time frame: Up to 5 years

Sponsors and collaborators

Lead sponsor

Adrenas Therapeutics Inc

Industry

Registry information

Official study 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

Important dates

Study start: 2021
Primary completion: 2024
Study completion: 2024
First posted: Nov 1, 2021
Registry last updated: Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed