Combining Pirfenidone With Mycophenolate Mofetil for Systemic Sclerosis-Related Interstitial Lung Disease (Scleroderma Lung Study III): An Investigator-Initiated, Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial.

ABSTRACT:

Objective

Mycophenolate mofetil (MMF) can stabilize or improve lung function in systemic sclerosis-related interstitial lung disease (SSc-ILD). We hypothesized that combining MMF with pirfenidone (PFD) would produce a significantly more rapid and/or greater improvement in lung function.

Methods

A randomized (1:1), double-blind, placebo (PLA)-controlled phase 2 trial was conducted in SSc-ILD in which patients received PFD or PLA (801 mg three times daily) along with MMF (1,500 mg twice daily) for 18 months. The primary outcome was change in percent predicted forced vital capacity (FVC-%). Linear mixed-effects models assessed treatment differences in a modified intention-to-treat population.

Results

Only fifty-one of 150 intended subjects (34%) were randomized (MMF+PLA,

SUBMITTER: Khanna D 

PROVIDER: S-EPMC12648624 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature