A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 103
- 试验地点
- 2
- 主要终点
- Change From Baseline in Serum Androstenedione at Week 4
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
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.
干预措施: Crinecerfont (Drug)
Placebo
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
干预措施: Crinecerfont (Drug)
Placebo
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Serum Androstenedione at Week 4
时间窗: 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.
次要结局
- 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)