PSSc-001 (LOTUSS) This study is a Phase 2, multinational, open-label, randomized, parallel-group, safety and tolerability study of pirfenidone in participants with systemic sclerosis-related interstitial lung disease (SSc-ILD).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
63
Pirfenidone will be administered orally at a dose of 267 mg one oral capsule TID (801 mg/day) for 1 or 2 weeks followed by two 267 mg oral capsules TID (1602 mg/day) for 1 or 2 weeks (titration period) and then three 267 mg oral capsules TID (2403 mg/day) for 12 weeks (maintenance period).
Mayo Clinic, Scottsdale
Scottsdale, Arizona, United States
Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
Percentage of participants who had treatment-emergent AEs, defined as newly occurring or worsening after first dose. Relatedness to (study drug) was assessed by the investigator (Yes/No). Participants with multiple occurrences of an AE within a category were counted once within the category.
Time frame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state.
Time frame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
University of California at Los Angeles (UCLA) Scleroderma Clinical Trial Consortium (SCTC) Gastrointestinal Trial (GIT) Questionnaire Scale Scores
UCLA SCTC GIT Scale 2.0 is a 34-item self-administered questionnaire to obtain participant's assessment of the frequency of GI symptoms in preceding 7 days and how symptoms affected his/her life. All but 2 items were scored on a 0 to 3 scale (0=better health, 3=worse health); remaining 2 items were scored as 0 (better health) and 1 (worse health). The 34 items are divided into seven scales (reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being, and constipation). Individual scale score was calculated as the average of the items in the scale. Individual scale score ranged from 0 to 3 for reflux, distention/bloating, fecal soilage, social functioning, and emotional well-being; 0 to 2 for diarrhea; and 0 to 2.5 for constipation. A total score was also calculated as the average of 6 of the 7 scales (omitting constipation) and ranged from 0 to 2.83. For individual and total scores 0 indicated better health and higher score indicates worse health.
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University of California, Los Angeles
Los Angeles, California, United States
Stanford University School of Medicine
Redwood City, California, United States
University of California, San Francisco
San Francisco, California, United States
National Jewish Medical and Research Center
Denver, Colorado, United States
Georgetown University Hospital
Washington D.C., District of Columbia, United States
Northwestern University, Chicago
Chicago, Illinois, United States
Boston University Medical Center
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Hospital for Special Surgery
New York, New York, United States
...and 12 more locations
Time frame: Baseline, Weeks 4, 8, 12, and 16