Description
The primary purpose of the CAHptain study is to explore the safety and efficacy of tildacerfont in children and adolescents aged 2 to 17 years of age with classic CAH, and evaluate if it may improve hormonal balance and allow children to take lower doses of glucocorticoids.
What to expect
The study drug will be evaluated in children and adolescents between 2 and 17 years of age who have classic CAH. Participants will receive tildacerfont tablets, to be taken daily. The tablets can be swallowed, crushed, or split. Visits and laboratory tests (blood and/or urine) will be done regularly during the study to monitor the safety of your treatment. The main part of the study is 12 weeks long but there is an optional extension where tildacerfont is given up to 84 more weeks. The commitment for this study includes 7 total visits, or 12 weeks total if participants decide to participate in the optional extension period. The study screening period can take up to 2 weeks to make sure all requirements necessary to participate are met. Then, the main study period consists of 12 weeks to determine how the study drug affects hormone levels and symptoms. After the main study period, there is an optional treatment extension period to continue monitoring the long-term effects of the study drug on hormone levels and symptoms for up to 2 years. A follow-up visit is scheduled 30 days after the last dose to perform a final health check after participants stop taking the study drug. Taking part in this study is completely voluntary, and you may choose to stop at any time.
