A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult 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
- 182
- 试验地点
- 71
- 主要终点
- Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24
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 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active 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 20 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
- 18 Years 至 —（Adult, Older Adult）
- 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.
- •Participants of childbearing potential must agree to use an acceptable method of contraception during the study.
排除标准
- •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 receptor 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.
研究组 & 干预措施
Placebo
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
干预措施: Placebo (Drug)
Placebo
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
干预措施: Crinecerfont (Drug)
Crinecerfont
Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.
干预措施: Crinecerfont (Drug)
结局指标
主要结局
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24
时间窗: Baseline, Week 24
Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
次要结局
- Change From Baseline in Glucose Tolerance at Week 24(Baseline, Week 24)
- Change From Baseline in Waist Circumference at Week 24(Baseline, Week 24)
- Change From Baseline in Serum Androstenedione at Week 4(Baseline, Week 4)
- Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24(Week 24)
- Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24(Baseline, Week 24)
- Percent Change From Baseline in Body Weight at Week 24(Baseline, Week 24)
- Change From Baseline in Percent Total Fat Mass at Week 24(Baseline, Week 24)
- Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4(Baseline, Week 4)
- Change From Baseline in Blood Pressure at Week 24(Baseline, Week 24)
- Change From Baseline in Menstrual Regularity at Week 24(Baseline, Week 24)
- Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24(Baseline, Week 24)