Safety and Tolerability of Pirfenidone in Participants With Systemic Sclerosis-Related Interstitial Lung Disease (SSc-ILD) (LOTUSS) (LOTUSS)
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ClinicalTrials.gov Identifier: NCT01933334 |
Recruitment Status :
Completed
First Posted : September 2, 2013
Results First Posted : September 28, 2015
Last Update Posted : August 4, 2016
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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).
Condition or disease | Intervention/treatment | Phase |
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Systemic Sclerosis | Drug: Pirfenidone | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 63 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 Start Date : | October 2013 |
Actual Primary Completion Date : | September 2014 |
Actual Study Completion Date : | September 2014 |

Arm | Intervention/treatment |
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Experimental: Pirfenidone: 4-Week Titration Group
Participants will receive one 267 milligrams (mg) oral pirfenidone capsule three times daily (TID) (801 mg per day [mg/day]) for 2 weeks followed by two 267 mg oral pirfenidone capsules TID (1602 mg/day) for 2 weeks (titration period) and then three 267 mg oral pirfenidone capsules TID (2403 mg/day) for 12 weeks maintenance period).
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Drug: Pirfenidone
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). |
Experimental: Pirfenidone: 2-Week Titration Group
Participants will receive one 267 mg oral pirfenidone capsule TID (801 mg/day) for 1 week followed by two 267 mg oral pirfenidone capsules TID (1602 mg/day) for 1 week (titration period) and then three 267 mg oral pirfenidone capsules TID (2403 mg/day) for 14 weeks (maintenance period).
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Drug: Pirfenidone
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). |
- Percentage of Participants With Treatment-Emergent Adverse Events (AEs) [ Time Frame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16) ]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.
- Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: From baseline up to 28 days after the last dose of study drug (last dose = Week 16) ]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.
- University of California at Los Angeles (UCLA) Scleroderma Clinical Trial Consortium (SCTC) Gastrointestinal Trial (GIT) Questionnaire Scale Scores [ Time Frame: Baseline, Weeks 4, 8, 12, and 16 ]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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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
- Diagnosis of SSc-ILD based on an high-resolution computed tomography (HRCT) scan
- 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
- At study entry, the participant either was not taking SSc-ILD medication or was taking cyclophosphamide or mycophenolate
Exclusion Criteria:
- Clinically significant pulmonary hypertension
- Known underlying liver disease
- Clinical evidence of significant aspiration or uncontrolled gastroesophageal reflux
- History of clinically significant asthma or chronic obstructive pulmonary disease
- Active infection
- Diagnosis of another connective tissue disorder
- Evidence of a malignancy that is likely to result in significant disability or require significant medical or surgical intervention
- History of unstable or deteriorating cardiac or pulmonary disease (other than SSc-ILD)
- Pregnancy or lactation
- Creatinine clearance <40 milliliters per minute (mL/min)
- Prior use of pirfenidone
- Unsuitable for enrollment or unlikely to comply with study requirements

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01933334

Study Chair: | For additional information, call InterMune Medical Information Telephone: 1-888-486-6411 | University of Cincinnati |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Genentech, Inc. |
ClinicalTrials.gov Identifier: | NCT01933334 |
Other Study ID Numbers: |
PSSc-001 |
First Posted: | September 2, 2013 Key Record Dates |
Results First Posted: | September 28, 2015 |
Last Update Posted: | August 4, 2016 |
Last Verified: | July 2016 |
pirfenidone SSc-ILD scleroderma systemic sclerosis interstitial lung disease |
Lung Diseases Lung Diseases, Interstitial Scleroderma, Systemic Scleroderma, Diffuse Sclerosis Pathologic Processes Respiratory Tract Diseases Connective Tissue Diseases Skin Diseases Pirfenidone |
Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Anti-Inflammatory Agents Antirheumatic Agents Antineoplastic Agents |