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Systemic SclerosisPhase 2LOTUSSNCT01933334

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

A Phase 2 study testing Pirfenidone in people with Systemic Sclerosis, run by Genentech, Inc. at 22 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Systemic Sclerosis
What is being tested?
Pirfenidone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
63 participants
Where
22 sitesin 3 countries
Study dates
Oct 2013to Sep 2014
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 12Show fewer
  • 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

Trial locations and contacts

22 sites in 3 countries.

22 sites in 3 countries

United States18 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Boston University Medical Center

    Boston, Massachusetts, 02118

  • Medical University South Carolina

    Charleston, South Carolina, 29425

  • Northwestern University, Chicago

    Chicago, Illinois, 60611

  • University of Cincinnati

    Cincinnati, Ohio, 45267

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • National Jewish Medical and Research Center

    Denver, Colorado, 80206

  • University of Texas, Houston

    Houston, Texas, 77030

  • University of California, Los Angeles

    Los Angeles, California, 90095

  • Hospital for Special Surgery

    New York, New York, 10021

  • Columbia University

    New York, New York, 10032

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15261

  • Stanford University School of Medicine

    Redwood City, California, 94063

  • University of Utah

    Salt Lake City, Utah, 84132

  • University of California, San Francisco

    San Francisco, California, 94143

  • Mayo Clinic, Scottsdale

    Scottsdale, Arizona, 85259

  • University of Toledo

    Toledo, Ohio, 43614

  • Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

Other trials in United States

Canada2 sites

  • St. Joseph's Healthcare

    Hamilton, Ontario, L8N 142

  • Toronto General Hospital

    Toronto, Ontario, M5T 3L9

Other trials in Canada

Italy2 sites

  • University of Florence

    Florence, 50139

  • Università di Torino

    Orbassano, Turin, 10043

Other trials in Italy

About this study

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)

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).

Being tested
Pirfenidone
Keywords
pirfenidone, SSc-ILD, scleroderma, systemic sclerosis, interstitial lung disease
Medical subject headings
Scleroderma, Systemic, Scleroderma, Diffuse, Lung Diseases, Interstitial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PirfenidoneDrug

    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).

Study groups in NCT01933334
Study groupRoleWhat they receive
Pirfenidone: 4-Week Titration GroupExperimentalParticipants 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).
Receives: Drug: Pirfenidone
Pirfenidone: 2-Week Titration GroupExperimentalParticipants 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).
Receives: Drug: Pirfenidone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)

  2. 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.

    Measured: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)

Secondary outcomes (1)

  1. 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.

    Measured: Baseline, Weeks 4, 8, 12, and 16

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Sep 2013 (estimated)
  2. Study startOct 2013
  3. Primary completionSep 2014
  4. Study completionSep 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
63 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01933334
Sponsor's protocol ID
PSSc-001
Acronym
LOTUSS
Status last verified
Jul 2016
First submitted
23 Aug 2013
Last updated
4 Aug 2016
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 7378088

  • 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.

    PubMed 27370878

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pirfenidone in people with Systemic Sclerosis, run by Genentech, Inc. at 22 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Canada, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Sep 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01933334, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Aug 2016.

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