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

About

Brief Summary

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).

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 2

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

18 Years

Maximum Age

N/A

Inclusion Criteria:

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

Exclusion Criteria:

≥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).

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