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).
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
≥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.
Parallel
LIQ861 (treprostinil) inhalation powder - Dose Titration Arm
Combination productTreprostinil 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
LIQ861 (treprostinil) inhalation powder - fixed dose arm
Combination productTreprostinil 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
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
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