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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
Sponsor:
Information provided by (Responsible Party):
Genentech, Inc.

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).


Condition or disease Intervention/treatment Phase
Systemic Sclerosis Drug: Pirfenidone Phase 2

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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
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).
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).
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).




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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
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

Information from the National Library of Medicine

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


  Show 22 Study Locations
Sponsors and Collaborators
Genentech, Inc.
Investigators
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Study Chair: For additional information, call InterMune Medical Information Telephone: 1-888-486-6411 University of Cincinnati

Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Genentech, Inc.
ClinicalTrials.gov Identifier: NCT01933334     History of Changes
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

Keywords provided by Genentech, Inc.:
pirfenidone
SSc-ILD
scleroderma
systemic sclerosis
interstitial lung disease

Additional relevant MeSH terms:
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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