A Study of LIQ861 (Inhaled Treprostinil) in Adults With Systemic Sclerosis Who Have Symptomatic Raynaud's Phenomenon Attacks

Официальное название

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

Обзор

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).

Вмешательства

• Комбинированный продукт LIQ861 (treprostinil) inhalation powder - Dose Titration Arm
  Treprostinil inhalation powder supplied as capsules in strengths of 26.5, 53, 79.5, and 106 mcg, administered by oral inhalation QID (approximately 3-4 hours apart during waking hours) for 21 days using the RS00 Model 8 Monodose DPI
• Комбинированный продукт LIQ861 (treprostinil) inhalation powder - fixed dose arm
  Treprostinil inhalation powder supplied as 26.5 mcg capsules administered by oral inhalation QID (approximately 3-4 hours apart during waking hours) for 21 days using the RS00 Model 8 Monodose DPI

Первичные конечные точки

• 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 [Срок оценки: 21 days]

Критерии участия

Критерии включения

• 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

Критерии исключения

• ≥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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07748000 · LTI-202

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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