Clinical trial NCT01933334: Safety and Tolerability of Pirfenidone in Participants With Systemic Sclerosis-Related Interstitial Lung Disease (SSc-ILD) (LOTUSS)
Official title: The LOTUSS Trial: An Open-Label, Randomized, Phase 2 Study of the Safety and Tolerability of Pirfenidone When Administered to Patients With Systemic Sclerosis-Related Interstitial Lung Disease (SSc-ILD) (LOTUSS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Systemic Sclerosis
Keywords: pirfenidone
SSc-ILD
scleroderma
systemic sclerosis
interstitial lung disease
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2013-09-02
Enrollment: 63
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: Inclusion Criteria:

1. Diagnosis of systemic sclerosis-related (SSc) confirmed by the American College of Rheumatology classification criteria of systemic sclerosis (Masi 1980); duration of diagnosis less than (\<) 7 years
2. Diagnosis of SSc-ILD based on an high-resolution computed tomography (HRCT) scan
3. Screening forced vital capacity (FVC) greater than equal to (\>=) 50 percent (%) of the predicted value, and screening carbon monoxide diffusing capacity (DLCO) \>=40% of the predicted value
4. At study entry, the participant either was not taking SSc-ILD medication or was taking cyclophosphamide or mycophenolate

Exclusion Criteria:

1. Clinically significant pulmonary hypertension
2. Known underlying liver disease
3. Clinical evidence of significant aspiration or uncontrolled gastroesophageal reflux
4. History of clinically significant asthma or chronic obstructive pulmonary disease
5. Active infection
6. Diagnosis of another connective tissue disorder
7. Evidence of a malignancy that is likely to result in significant disability or require significant medical or surgical intervention
8. History of unstable or deteriorating cardiac or pulmonary disease (other than SSc-ILD)
9. Pregnancy or lactation
10. Creatinine clearance \<40 milliliters per minute (mL/min)
11. Prior use of pirfenidone
12. Unsuitable for enrollment or unlikely to comply with study requirements

Primary outcomes:
measure: Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
timeFrame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
measure: Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
timeFrame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
Secondary outcomes:
measure: University of California at Los Angeles (UCLA) Scleroderma Clinical Trial Consortium (SCTC) Gastrointestinal Trial (GIT) Questionnaire Scale Scores
timeFrame: Baseline, Weeks 4, 8, 12, and 16
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic, Scottsdale
state: Arizona
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Redwood City
country: United States
facility: Stanford University School of Medicine
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Denver
country: United States
facility: National Jewish Medical and Research Center
state: Colorado
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Chicago
country: United States
facility: Northwestern University, Chicago
state: Illinois
city: Boston
country: United States
facility: Boston University Medical Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: New York
country: United States
facility: Columbia University
state: New York
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Toledo
country: United States
facility: University of Toledo
state: Ohio
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University South Carolina
state: South Carolina
city: Houston
country: United States
facility: University of Texas, Houston
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Hamilton
country: Canada
facility: St. Joseph's Healthcare
state: Ontario
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Orbassano
country: Italy
facility: Università di Torino
state: Turin
city: Firenze
country: Italy
facility: University of Florence
Study officials:
affiliation: University of Cincinnati
name: For additional information, call InterMune Medical Information Telephone: 1-888-486-6411
role: STUDY_CHAIR
References:
citation: Preliminary criteria for the classification of systemic sclerosis (scleroderma). Subcommittee for scleroderma criteria of the American Rheumatism Association Diagnostic and Therapeutic Criteria Committee. Arthritis Rheum. 1980 May;23(5):581-90. doi: 10.1002/art.1780230510.
citation: Khanna D, Albera C, Fischer A, Khalidi N, Raghu G, Chung L, Chen D, Schiopu E, Tagliaferri M, Seibold JR, Gorina E. An Open-label, Phase II Study of the Safety and Tolerability of Pirfenidone in Patients with Scleroderma-associated Interstitial Lung Disease: the LOTUSS Trial. J Rheumatol. 2016 Sep;43(9):1672-9. doi: 10.3899/jrheum.151322. Epub 2016 Jul 1.