A Study of LIQ861 (Inhaled Treprostinil) in Adults With Systemic Sclerosis Who Have Symptomatic Raynaud's Phenomenon Attacks | NCT07748000

NCT07748000

Overview

NCT ID: NCT07748000
Lead Sponsor: Liquidia Technologies, Inc.INDUSTRY
Official Title: 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
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).
Conditions

Ages Eligible for Study: Adult (18-64)Older Adult (65+)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
Eligibility Criteria: 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).

Central Contacts: Liquidia Point of ContactContact
LiquidiaStudies@liquidia.com

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