Scleroderma Lung Study III - Combining Pirfenidone With Mycophenolate

Completed · Phase 2 · Has a placebo group

Conditions studied: Scleroderma, Systemic, Interstitial Lung Disease

In brief

A Phase II multi-center, double-blind, parallel group, randomized and placebo-controlled clinical trial addressing the treatment of patients with active and symptomatic Scleroderma-related interstitial lung disease (SSc-ILD).

Key facts

Study ID

NCT03221257

Run by

Michael Roth

People needed

51

Starts

2017-11-28

Expected to finish

2022-06-13

Last updated by the study team

2023-12-15

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age ≥18 yrs
• Scleroderma as determined by the 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria.
• Grade ≥2 on the Magnitude of Task component of the Mahler Modified Dyspnea Index
• FVC-% of ≤85% at screening
• Onset of the first non-Raynaud manifestation of SSc within the prior 84 months.
• Presence of any ground-glass opacification (GGO) on thoracic high-resolution computed tomography (HRCT)
• Repeat FVC-% at the baseline visit within 10% of the FVC-% value measured at screening. If these criteria are not met, a repeat FVC-% may be obtained within 7 days and the subject may qualify for randomization if the repeat FVC-% agrees within 10% of the FVC-% obtained at screening.

You may not qualify if…

• Disease features supporting the primary diagnosis of another connective tissue disease such as rheumatoid arthritis, systemic lupus erythematosus or mixed connective tissue disease (Features consistent with a secondary Sjogren syndrome or scleroderma-associated myopathy will be allowed).
• FVC-% of <45% at either screening or baseline.
• Forced Expiratory Volume in the first second (FEV1)/Forced Vital Capacity (FVC) ratio <0.65 at either screening or baseline.
• Diffusing capacity of the lung for carbon monoxide adjusted for hemoglobin, expressed as a percentage of the normal predicted value (DLCOHb-%) of <30% at screening or <25% at baseline.
• a) All participants with a DLCOHb-% between 30 to 40% must have pulmonary artery pressures documented by either echocardiogram, right heart catheterization or magnetic resonance imaging in order to be considered for inclusion.
• Diagnosis of clinically significant resting pulmonary hypertension requiring treatment or mild pulmonary hypertension requiring treatment with more than one oral medication as ascertained prior to study evaluation or as part of a standard of care clinical assessment performed outside of the study protocol.
• Evidence of uncontrolled congestive heart failure, unstable ischemic heart disease, history of complicated pulmonary embolism impacting on heart or lung function, or unstable cardiac arrhythmia requiring chronic anticoagulation.
• Clinically significant abnormalities on HRCT not attributable to SSc
• Hematologic abnormality at screening including:
• Leukopenia (white blood cells [WBC] <4.0x10\^3/µl).
• Thrombocytopenia (platelet count <120.0x10\^3/µl).
• Clinically significant anemia [Hemoglobin (Hgb) <10.0 g/dl].
• Participants with an identified and correctable etiology may be eligible if repeat testing within the maximal 90-day screening period meets all criteria.
• A diagnosis of chronic liver disease or abnormal baseline liver function test (LFTs) or total bilirubin that are >2.0 x upper normal limit
• Serum creatinine >2.0mg/dl
• History of recurrent aspiration, uncontrolled heartburn, or gastroesophageal reflux disease (GERD) with a reflux scale score of >1.00 as determined by a UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Scale (UCLA SCTC GIT), Version 2.0.
• Participants with uncontrolled heartburn or GERD that is amenable to medical management may be eligible if repeat testing within the maximal 90-day screening period meets this criteria.
• Known achalasia, esophageal stricture or esophageal dysfunction sufficient to limit the ability to swallow medication.
• Pregnancy (as documented by blood test) and/or breast feeding
• If of child bearing potential (a female participant < 55 years of age who has not been postmenopausal for ≥ 5 years or who has not had a bilateral salpingectomy, hysterectomy and/or oophorectomy), failure to employ two reliable means of contraception which may include surgical sterilization, barrier methods, spermicides, intrauterine devices, and/or hormonal contraception, unless the participant chooses abstinence (to avoid heterosexual intercourse completely). If a subject chooses abstinence, then a second reliable means of contraception is not needed.
• Prior use of potential disease modifying antirheumatic drugs (DMARDs) according to the following exposure rules:
• Use of oral cyclophosphamide (CYC), MMF, azathioprine or other oral or short half-life DMARDs (as detailed in Protocol Section 7.5.1a) for more than 6 months in the past year as determined at the time of the initial screening visit.
• Treatment with more than three intravenous doses of CYC, one treatment course of Rituximab or other intravenous or injectable DMARDs (as detailed in Protocol Section 7.5.1b) in the past year.
• More distant history of treatment with a DMARD is allowed as long as the patient has a new diagnosis/new episode of active SSc-ILD since stopping that treatment and meets the criteria noted in 15a or 15b.
• Use of CYC, MMF, azathioprine, Rituximab or other DMARD (as defined in Protocol Section 7.5.1a\&b) in the 30 days prior to their baseline visit unless the patient is on MMF and the responsible physician indicates that continued use is in the best clinical interest of the patient.

Where it is running

• University of California Los Angeles — Los Angeles, California, United States
• University of Colorado — Aurora, Colorado, United States
• Georgetown University — Washington D.C., District of Columbia, United States
• Northwestern University — Chicago, Illinois, United States
• Indiana University Health — Indianapolis, Indiana, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Harvard Medical School, Brigham & Women's Hospital — Boston, Massachusetts, United States
• Boston University, School of Medicine — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Rutgers University — New Brunswick, New Jersey, United States
• Hospital for Special Surgery — New York, New York, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• University of Texas Medical School at Houston — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• University of Washington Medical Center — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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