Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Clas… (NCT04806451) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
United States103 participantsStarted 2021-06-25
Plain-language summary
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
Who can participate
Age range2 Years – 17 Years
SexALL
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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 CAH due to 21-hydroxylase deficiency.
* Be on a stable steroid regimen.
* Have elevated androgen levels.
* Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.
Exclusion Criteria:
* Have a diagnosis of any of the other 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 to be cured.
* Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).
* Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.
* Have received an 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 substance (drug) or alcohol abuse.
* Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.
What they're measuring
1
Change From Baseline in Serum Androstenedione at Week 4