A Phase 2a Randomized, Open-Label, Dose-Finding Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of LIQ861 in Participants With Systemic Sclerosis Experiencing Symptomatic Raynaud's Phenomenon Attacks / RE-WARM
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 75
- 主要终点
- Participant incidence of tolerated dose(s) of LIQ861 defined as a dose level or dose regimen at which ≥80% of participants complete the Treatment Period with ≥80% compliance without permanent discontinuation of LIQ861 due to a TEAE
研究概览
简要总结
Systemic sclerosis (SSc) is a rare autoimmune disease in which many patients experience Raynaud's phenomenon (RP) - painful episodes where commonly fingers or toes change color and become painful, numb, or tingly, usually triggered by cold or stress. This open-label study is testing an inhaled powder form of treprostinil (LIQ861) to find out which doses can be taken safely and comfortably by adults with SSc who have frequent symptomatic RP attacks. About 75 adults will be randomly assigned (2 to 1) to either a dose-titration group or a fixed low-dose group. The study also explores whether treatment reduces the number, severity, and impact of RP attacks. The main goal is to identify tolerated dose(s).
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
- 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
- •Male or female ≥18 years of age
- •Classification of SSc per the 2013 ACR/EULAR criteria.
- •Diagnosis or history of Raynaud's phenomenon
- •Minimum of 12 (maximum 100) symptomatic RP attacks documented in the ePRO diary over ≥4 separate days
排除标准
- •≥3 digital ulcers at screening; a new digital ulcer between screening and randomization; gangrene or amputation within 6 months; digital ulcer infection within 3 months.
- •History of cervical/digital sympathectomy or hand botulinum toxin injections within 90 days (or planned during the study).
- •Clinically significant bleeding risk, coagulation disorder, or platelet count <100 x 10^9/L.
- •Known WHO Group 1-5 pulmonary hypertension; significant obstructive (FEV1 <65%) or restrictive (FVC <60%) lung disease; need for supplemental oxygen; severe asthma.
- •Severe uncontrolled hypertension (SBP ≥160 and/or DBP >100 mmHg) or severe hypotension (SBP <85 mmHg).
- •Any parenteral, inhaled, or oral prostacyclin or prostacyclin receptor agonist within 12 weeks of screening; initiation/dose change of vasodilators within 4 weeks of screening; non-topical CYP2C8 inhibitors/inducers (unless discontinued for >5 half-lives before randomization).
研究组 & 干预措施
Arm 1: LIQ861 Dose Titration Arm
干预措施: LIQ861 (treprostinil) inhalation powder - Dose Titration Arm (Combination Product)
Arm 2: LIQ861 Fixed Dose
干预措施: LIQ861 (treprostinil) inhalation powder - fixed dose arm (Combination Product)
结局指标
主要结局
Participant incidence of tolerated dose(s) of LIQ861 defined as a dose level or dose regimen at which ≥80% of participants complete the Treatment Period with ≥80% compliance without permanent discontinuation of LIQ861 due to a TEAE
时间窗: 21 days
次要结局
未报告次要终点