A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
研究概览
简要总结
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
详细描述
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects. To be eligible for this study, an individual must either have completed Study SPR001-201 or meet eligibility criteria for SPR001-naïve subjects. The expected duration of study participation for each subject is up to approximately 5 months. This includes a screening period of ≤30 days, a treatment period of 12 weeks, and a safety follow-up period of 30 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Is approved by the Sponsor's Medical Monitor
- •Is on a stable regimen of glucocorticoid replacement for ≥30 days before baseline that is expected to remain stable throughout the study
- •If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will have serum 17-OHP measured at screening.
- •Agrees to follow contraception guidelines
- •Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol
排除标准
- •Experienced a clinically significant AE considered at least possibly related to SPR001 in Study SPR001-201
- •If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will be screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening
- •Is at increased risk of suicide
- •Clinically significant depression or anxiety at screening or baseline
- •Clinically significant abnormal clinical or laboratory assessments must be discussed with the Medical Monitor to determine eligibility for this study.
- •Subjects who routinely work overnight shifts require Medical Monitor approval for enrollment
- •Females who are pregnant or lactating
- •Use of any other investigational drug within 30 days or 5 half-lives before screening
- •Use of prohibited concomitant medications (including rosiglitazone, testosterone, and strong inhibitors and/or inducers of CYP3A4) within 30 days or 5 half-lives of baseline. Medications metabolized by CYP3A4, 2C8, 2C9, or 2C19, especially those that are sensitive substrates or substrates with narrow therapeutic ranges should be discussed on a case-by-case basis with the Medical Monitor.
研究组 & 干预措施
SPR001
SPR001 at Dose A
干预措施: SPR001 (Drug)
结局指标
主要结局
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
时间窗: Over 12 weeks
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
次要结局
- Change From Baseline in 17-hydroxyprogesterone (17-OHP)(Week 12)
- Change From Baseline in Androstenedione (A4)(Week 12)
- Change From Baseline in Adrenocorticotropic Hormone (ACTH)(Week 12)