Safety and Tolerability of Pirfenidone in Patients With Systemic Sclerosis−Related Interstitial Lung Disease (SSc-ILD) (LOTUSS)

Systemic Sclerosis Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01933334
• Other study ID #: PSSc-001
• Status: Completed
• Phase: Phase 2
• First received: August 23, 2013
• Last updated: August 25, 2015
• Start date: September 2013
• Est. completion date: September 2014

Study information

• Verified date: August 2015
• Source: Genentech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health CanadaItaly: Agenzia Italiana del Farmaco
• Study type: Interventional

Clinical Trial Summary

PSSc-001 (LOTUSS)

This study is a Phase 2, multinational, open-label, randomized, parallel-group, safety and tolerability study of pirfenidone in patients with systemic sclerosis−related interstitial lung disease (SSc-ILD).

Description:

This study is a Phase 2, multinational, open-label, randomized, parallel-group, safety and tolerability study of pirfenidone in patients with systemic sclerosis−related interstitial lung disease (SSc-ILD)

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: September 2014
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Diagnosis of SSc confirmed by the American College of Rheumatology classification criteria of systemic sclerosis (Masi 1980); duration of diagnosis <7 years

2. Diagnosis of SSc-ILD based on an HRCT scan

3. Screening FVC =50% of the predicted value, and screening DLCO =40% of the predicted value

4. At study entry, the patient either is not taking SSc-ILD medication or is taking cyclophosphamide or mycophenolate mofetil

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 mL/min

11. Prior use of pirfenidone

12. Unsuitable for enrollment or unlikely to comply with study requirements.

Study Design

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Lung Diseases
• Lung Diseases, Interstitial
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis
• Systemic Sclerosis

Intervention

Drug:
• Pirfenidone
antifibrotic drug

Locations

Country	Name	City	State
Canada	St. Joseph's Healthcare	Hamilton	Ontario
Canada	Toronto General Hospital	Toronto	Ontario
Italy	University of Florence	Firenze
Italy	Università di Torino	Orbassano	Turin
United States	University of Michigan	Ann Arbor	Michigan
United States	Boston University Medical Center	Boston	Massachusetts
United States	Medical University South Carolina	Charleston	South Carolina
United States	Northwestern University, Chicago	Chicago	Illinois
United States	University of Cincinnati	Cincinnati	Ohio
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	National Jewish Medical and Research Center	Denver	Colorado
United States	University of Texas, Houston	Houston	Texas
United States	University of California, Los Angeles	Los Angeles	California
United States	Columbia University	New York	New York
United States	Hospital for Special Surgery	New York	New York
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Stanford University School of Medicine	Redwood City	California
United States	University of Utah	Salt Lake City	Utah
United States	University of California, San Francisco	San Francisco	California
United States	Mayo Clinic, Scottsdale	Scottsdale	Arizona
United States	University of Toledo	Toledo	Ohio
United States	Georgetown University Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Italy, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	From baseline up to 28 days after the last dose of study drug (last dose = Week 16)	No
Primary	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.	From baseline up to 28 days after the last dose of study drug (last dose = Week 16)	No
Secondary	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.	Baseline, Weeks 4, 8, 12, and 16	No