A Multicenter, Open-label Extension Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Amgen
- 入组人数
- 174
- 试验地点
- 130
- 主要终点
- Change From Baseline in Forced Vital Capacity Percentage (FVC%) Predicted at Week 52
研究概览
简要总结
Primary Objectives:
- The primary efficacy objective is to assess the efficacy of 52 weeks of open-label treatment with HZN-825 in participants with diffuse cutaneous systemic sclerosis, as measured by change from both baselines in forced vital capacity percent (FVC %) predicted.
- The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN-825, inclusive of, but not limited to, adverse events (AEs), serious AEs (SAEs) and the adverse event of special interest (AESI), from Day 1 to 4 weeks after last dose.
详细描述
This is an open-label, repeat-dose, multicenter extension trial of HZNP-HZN-825-301. Participants who complete the double-blind Treatment Period (Week 52) in Trial HZNP-HZN-825-301 will be eligible to enter this 52-week extension trial. Participants entering this extension trial will complete the Week 52 Visit activities in HZNP-HZN-825-301 and will not complete the Safety Follow-up Visit 4 weeks after the last dose of trial drug in HZNP-HZN-825-301.
Acquired from Horizon in 2024.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Completed the double-blind Treatment Period (Week 52) in Trial HZNP-HZN-825-301; participants prematurely discontinued from trial drug in Trial HZNP-HZN-825-301 for reasons other than safety or toxicity can be included at the discretion of the Investigator after completing Trial HZNP-HZN-825-301 scheduled visits, including Week 52 assessments.
排除标准
- •Anticipated use of another investigational agent for any condition during the course of the trial.
- •New diagnosis of malignant condition after enrolling in Trial HZNP-HZN-825-301 (except successfully treated basal/squamous cell carcinoma of the skin or cervical cancer in situ).
- •Women of childbearing potential (WOCBP) or male participants not agreeing to use highly effective method(s) of birth control throughout the trial and for 4 weeks after last dose of trial drug as defined in the protocol.
- •Any new development with the participant's disease or condition or any significant laboratory test abnormality during the course of Trial HZNP-HZN-825-301 that, in the opinion of the Investigator, would potentially put the subject at unacceptable risk.
- •Pregnant or lactating women.
- •Participants will be ineligible if, in the opinion of the Investigator, they are unlikely to comply with the trial protocol or have a concomitant disease or condition that could interfere with the conduct of the trial.
研究组 & 干预措施
HZN-825
HZN-825 will be administered by mouth (PO) twice daily (BID) for 52 weeks
干预措施: HZN-825 (Drug)
结局指标
主要结局
Change From Baseline in Forced Vital Capacity Percentage (FVC%) Predicted at Week 52
时间窗: Baseline and Week 52
FVC is the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible, as measured by spirometry. FVC is a measure of respiratory function. FVC% predicted was calculated by taking the observed FVC measurement and dividing it by a predicted value multiplied by 100 (% FVC predicted = (FVC observed/FVC predicted) x 100). The predicted value is an average of the normal FVC volume for a person of the same sex, ethnicity, age and height.
Number of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
时间窗: From 1st dose to last dose + 28 days; median (min, max) time on trial was 8.5 (1.0, 14.0) months
An adverse event (AE) was defined as any untoward medical occurrence in a trial participant who received an investigational product (IP), regardless of a causal relationship with treatment. An AE could be any unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease temporally associated with the use of an IP. TEAEs were defined as events that began or worsened in severity on or after the first dose of treatment through 28 days after the last dose or the cutoff date for ongoing participants. Serious TEAEs were those that resulted in death, were life-threatening, required or prolonged hospitalization, caused significant disability/incapacity, led to a congenital anomaly/birth defect, or were considered other important medical events. Clinically significant changes in vital signs, electrocardiograms (ECGs), and laboratory tests were included as TEAEs.
Number of Participants Who Experienced AEs of Special Interest (AESI)
时间窗: Day 1 and at Weeks 4, 28 and 52
The following AESI was identified for this trial: Orthostatic hypotension defined as a reduction of systolic blood pressure by ≥20 mmHg or reduction of diastolic blood pressure by ≥10 mmHg and associated with symptoms such as lightheadedness, blurred vision, weakness, fatigue, cognitive impairment, nausea, palpitations, tremulousness, headache, presyncope or syncope.
Number of Participants Using Any Concomitant Medication
时间窗: From 1st dose to last dose + 28 days; median (min, max) time on trial was 8.5 (1.0, 14.0) months
Concomitant medications were defined as any medication that was ongoing, had a start date on or after the first dose of the trial drug, or had a stop date on or after the first dose date.
次要结局
未报告次要终点