A Prospective Phase II, Open-Label, Single-arm, Multicenter, Study to Assess Efficacy and Safety of SEG101 (Crizanlizumab), in Sickle Cell Disease Patients With Priapism (SPARTAN)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 17
- 主要终点
- Percent Change in Priapic Events From Baseline to 26 Weeks
研究概览
简要总结
The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.
详细描述
Before participating in this study, information to determine key eligibility criteria was collected as a part of a 14-week Pre-Screening period. The study included a 12-week Screening period and a 52-week (1 year) Treatment period. Eligible participants received crizanlizumab 5 mg/kg by intravenous infusion (IV). Study treatment was received at clinic visits on Week 1 Day 1, Week 3 Day 1, and then on Day 1 of every 4-week cycle. Efficacy assessments included evaluation of priapic and vaso-occlusive (VOC) events. Safety assessments included laboratory tests, electrocardiograms (ECGs), vital signs and physical examinations. Participants had a safety follow-up for up to 15 weeks after the last dose.
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
- 12 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients aged 12 years and above
- •Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
- •Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
- •Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
- •If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
- •If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
- •Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures
排除标准
- •Had penile prosthetic implants or shunts or any other surgical procedure on the penis performed within 12 months prior to consenting was not allowed
- •Took drugs/medications that may induce priapism over the 14 weeks preceding study entry
- •Received leuprolide acetate (Lupron) or any other gonadotropin releasing hormone receptor agonist agent within 3 months before pre-screening
- •Had an erection lasting more than 12 hours over the 14 weeks preceding study entry
- •Had an erection lasting more than 12 hours during the 12 weeks of the Screening period
研究组 & 干预措施
Crizanlizumab 5 mg/kg
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
干预措施: Crizanlizumab (Drug)
结局指标
主要结局
Percent Change in Priapic Events From Baseline to 26 Weeks
时间窗: Baseline up to 26 weeks
A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. Number of priapic events was summarized at Baseline (adjusted for 26 weeks) and by 26 weeks, and percent reduction from adjusted Baseline by 26 weeks was summarized.
次要结局
- Annualized Rate of Priapic Events(Baseline up to 26 and 52 weeks)
- Number of Acute Priapic Events From Baseline to 26 and 52 Weeks(Baseline up to 26 and 52 weeks)
- Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)(Baseline up to 26 and 52 weeks)
- Annualized Rate of Complicated VOCs(Baseline up to 26 and 52 weeks)