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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)

IRB Number: 13224

February 07, 2014

Institutional Review Board, Hospital for Special Surgery

The safety of our participants is our top priority. The trial/study is approved and periodically reviewed by the Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.

Before enrolling in a clinical trial or research study, the investigator will explain the purpose of the trial/study, its expected benefits, any possible risks or side effects, and what your role will be. If you want to join the trial/study, you must sign informed consent documents. You can leave a trial/study at any time without penalty.

For further information, see Understanding Clinical Trials/Research Studies.

Principal Investigator

Robert F. Spiera, MD



Jessica K. Gordon, MD
Joanna Harp, MD
Lindsay Lally, MD
Daniele Lerner, BA
Uzunma Udeh, BA
Nina Paddu, BA


5 patients will be recruited at HSS
Enrollment Period: will end in April 2014
Duration: 16 weeks

All subjects in the study will receive pirfenidone (up to 2403 mg per day). Pirfenidone will be supplied in white, opaque, hard gelatin capsules and will be taken, after step by step dose escalation is completed as 3 capsules by mouth, 3 times a day (a total of 9 capsules per day)

Eligible subjects in this study may be currently receiving cyclophosphamide, or mycophenolate mofetil (prescribed by their doctors), or no SSc-ILD treatment. In this study, all eligible participants will receive pirfenidone. Subjects will be assigned randomly (by chance) to one of two groups. In one group, subjects will gradually increase the dose of pirfenidone over 2 weeks to reach the full dose, which is 2403 mg/day. In the other group, subjects will gradually increase the dose of pirfenidone over 4 weeks to reach the full dose, which is 2403 mg/day.

Inclusion/Exclusion Criteria

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.

Contact Information

Daniele Lerner