Overview
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of treatment with crinecerfont. Duration of participation is approximately 20 months.
Eligibility
Inclusion Criteria:
- Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
- Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
- Be on a stable regimen of steroidal treatment for CAH.
- Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.
Exclusion Criteria:
- Have a diagnosis of any of the other known forms of classic CAH.
- Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
- Have a clinically significant unstable medical condition or chronic disease other than CAH.
- Have a history of cancer unless considered cured.
- Are pregnant.
- Have a known history of clinically significant arrhythmia or abnormalities on ECG.
- Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
- Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
- Have current substance dependence, or current substance (drug) or alcohol abuse.
- Have had a blood loss 550 mL or donated blood or blood products within 8 weeks prior to the study.