A Phase 2 Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety and Efficacy of Ifetroban in Patients With Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension (SSc-PAH)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 34
- 试验地点
- 24
- 主要终点
- Incidence of adverse events (AEs) and Serious AEs (SAEs)
研究概览
简要总结
The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH).
详细描述
This study is a randomized, placebo-controlled, double-blind phase 2 trial of patients with dcSSc or SSc-PAH. Twenty participants with SSc-PAH and 14 participants with dcSSc will be randomized to receive either oral ifetroban daily or matching placebo. Study participants will be treated for 12 months, followed by a 30-day follow-up period. The study will test whether ifetroban is safe and statistically superior to placebo in reducing the effects of their disease at month 12 and explore the ability of ifetroban to prevent or reverse progression in patients with early disease duration and reverse established disease in patients with longer disease duration.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diffuse Cutaneous Criterion:
- •1. Systematic Sclerosis (SSc), as defined using the 2013 American College of Rheumatology/ European Union League Against Rheumatism Classification Criteria and dcSSc within 7 years following initial diagnosis as defined by the onset of the first non-Raynaud symptom.
- •SSc-PAH Criteria:
- •Adults fulfilling the 2013 American College of Rheumatology/ European Union League Against Rheumatism Classification Criteria with confirmed SSc-PAH (limited or dcSSc) confirmed via previous cardiac catheterization
- •Stable oral therapy for PAH for at least 30 days (monotherapy or combination)
- •New York Heart Association (NYHA) Class I-III Heart Failure
排除标准
- •Have a diagnosis of systemic sclerosis sine scleroderma;
- •Be less than 18 years of age or greater than or equal to 80 years of age;
- •Be pregnant, nursing, or planning to become pregnant;
- •Current or planned treatment with prostanoid therapy;
- •Current or planned treatment with pirfenidone;
- •Use of rituximab in the last 3 months;
- •Use of mycophenolic acid (Myfortic, CellCept) at a stable dose for less than 3 months;
- •Current or planned corticosteroid therapy greater than 15mg per day of prednisone or prednisone equivalent;
- •Significant lung disease, defined as FVC < 50% predicted or DLCO <40% predicted;
- •Significant kidney disease, defined as Glomerular Filtration Rate (GFR) < 60 ml/min;
- •Have moderate or severe hepatic impairment;
- •Contraindication to MRI (e.g., implanted magnetic material, claustrophobia);
- •Known hypersensitivity to gadolinium;
- •Any cause of pulmonary hypertension other than World Health Organization (WHO) Group I associated with SSc;
- •Use of aspirin > 81 mg per day in the last two weeks;
- •Use of warfarin, heparin or other anticoagulants in the last 30 days;
- •Recent (within 6 weeks) myocardial infarction or persistent atrial arrhythmias;
- •Have a history of allergy or hypersensitivity to ifetroban;
- •Have taken investigational drugs within 30 days before study treatment administration;
- •Inability to understand the requirements of the study, inability to understand spoken English and abide by the study restrictions and to return for the required treatments and assessments;
- •Be otherwise unsuitable for the study, in the opinion of the investigator.
研究组 & 干预措施
Patients with SSc-PAH
Patients with SSc-PAH will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
干预措施: Oral Placebo (Drug)
Patients with dcSSc
Patients with dcSSc will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
干预措施: Oral Placebo (Drug)
Patients with SSc-PAH
Patients with SSc-PAH will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
干预措施: Oral Ifetroban (Drug)
Patients with dcSSc
Patients with dcSSc will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
干预措施: Oral Ifetroban (Drug)
结局指标
主要结局
Incidence of adverse events (AEs) and Serious AEs (SAEs)
时间窗: 56 weeks
Safety is measured using AEs, including clinical significant changes in vital signs, laboratory test abnormalities and clinical tolerability of ifetroban.
次要结局
- Change from baseline in the modified Rodnan skin score (mRSS)(Baseline, 12, 26, 39, and 52 weeks)
- Change from baseline in forced vital capacity (FVC)(Baseline, 12, 26, and 52 weeks)
- Change from baseline in diffusion capacity for carbon monoxide (DLCO)(Baseline, 12, 26, and 52 weeks)