Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Drug: Placebo
- Registration Number
- NCT04806451
- Lead Sponsor
- Neurocrine Biosciences
- Brief 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 103
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Crinecerfont Crinecerfont Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks. Placebo Crinecerfont Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks. Placebo Placebo Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
- Primary Outcome Measures
Name Time Method Change From Baseline in Serum Androstenedione at Week 4 Baseline, Week 4 Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 Baseline, Week 4 Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28 Baseline, Week 28 Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28 Week 28 Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28 Baseline, Week 28 Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28 Baseline, Week 28 Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28 Baseline, Week 28
Trial Locations
- Locations (2)
Neurocrine Clinical Site
🇬🇧London, United Kingdom
Neurocrine Clinical site
🇺🇸Saint Louis, Missouri, United States