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Autoantibody Reduction Therapy in Patients With Idiopathic Pulmonary Fibrosis

Phase 2
Completed
Conditions
Ambulatory IPF
Interventions
Drug: Placebo
Drug: Rituximab
Registration Number
NCT01969409
Lead Sponsor
University of Alabama at Birmingham
Brief Summary

Recent research studies have suggested that proteins called antibodies that are produced by the immune system might be involved in the lung damage of idiopathic pulmonary fibrosis (IPF). Antibodies produced by the immune system normal help to fight infections by attacking bacteria and viruses without harming our own tissues. In patients with IPF, there is evidence that certain antibodies (called autoantibodies) attack the lung and contributes to the injury and scarring that occurs in IPF. Our recent studies have found that many IPF patients appear to have excessive autoantibody levels in blood and lungs that might make their disease worse.

Rituximab is a medication approved by the Food and Drug Administration (FDA) for the treatment of autoantibody diseases such as rheumatoid arthritis. Rituximab works by destroying B cells, a type of white blood cell, called a B-lymphocyte, which produce autoantibodies. In this research study, rituximab will be given into a vein to reduce the autoantibody levels that we believe might be contributing to the lung damage in IPF.

This study is being conducted to determine if rituximab provides beneficial effects for IPF patients by decreasing further lung injury.

Detailed Description

This is a double-blinded, Phase II trial in which 58 ambulatory IPF patients at any of four medical centers (University of Pittsburgh, University of Chicago, Geisinger Medical Center, and Temple University) will be randomized equally to 1. placebo or 2. two doses of rituximab 1 gm i.v., with a 14 day interval inbetween doses.

Subjects will be followed for 9 months.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
58
Inclusion Criteria

Ambulatory patients with a diagnosis of IPF, not established >5 years from the enrollment date, that fulfills American Thoracic Society (ATS)/European Thoracic Society (ETS) Consensus Criteria.

Ability and willingness to give informed consent. Presence of autoantibodies against Hepatoma-2 (HEp-2) cells, the assay for the primary endpoint.

Age 50-85 y.o.

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Exclusion Criteria

Diagnoses of current infection, proven or suspected by participating physicians based upon their clinical assessments.

Presence of active hepatitis B or C, or HIV infection. Presence of positive CONVENTIONAL autoimmune serologic tests, e.g., Antinuclear Antibodies (ANA), Rheumatoid Factor (RF), Anti-Ro, Anti-LA, Anti-Ribonucleoprotein Antibodies (RNP), Anti-Jo-1.

History of reaction to murine-derived products or any of the trial medications, or prior exposures to human-murine chimeric antibodies.

Malignancy, excluding basal or squamous cell skin cancer and low-risk prostate cancer, defined as stage T1 or T2a with Prostate-Specific Antigen (PSA) less than 10 ng/dl.

Unwillingness to complete post-treatment surveillance for 9 months. Diagnosis of major morbidities (aside from IPF) expected to interfere with subjects' study participation for 9 months.

Treatment for >5 days within the preceding month with >10 mg. prednisone (or equivalent corticosteroid) or any treatment during the preceding month with a potent cellular immunosuppressant (e.g., cyclophosphamide, methotrexate, mycophenolate, azathioprine, calcineurin inhibitors, etc.).

Uncontrolled diabetes or hypertension that preclude safe treatment with methylprednisolone.

Concurrent participation in other experimental trials.

Pregnancy or unwillingness to use contraception during the duration of the study among female participants with child-bearing potential.

Ratio of forced expiratory volume in 1 second to forced vital capacity (FEV1/FVC) <70% of predicted values.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboThese subjects will receive i.v. placebo (5% dextrose in water) administered identically to the rituximab.
RituximabRituximabRituximab i.v. given on two occasions, with 14 days between doses.
Primary Outcome Measures
NameTimeMethod
Autoantibodies to Human Epidermoid (HEp)-2 Cellsbaseline to 9 months

Titers of anti-HEp-2 autoantibodies, by indirect immunofluorescence assays (IFA) over 9 months, month 9 reported. Titers represent the highest dilution of patient plasma wherein the autoantibodies can be detected. Higher titers denote greater autoantibody concentrations.

Secondary Outcome Measures
NameTimeMethod
Changes in Forced Vital Capacity (FVC)baseline thru 9 months

FVC values over the observation period will be measured by spirometry, month 9 reported.

Number of Acute Exacerbationsduring the study duration of 9 months

The number of acute exacerbations, defined using consensus criteria (new/worsened hypoxemia and dyspnea, with characteristic radiographic changes within the last 30 days).

Changes in Anti-Heat Shock Protein 70 (HSP70) Autoantibodiesbaseline to 9 months

Changes of anti-HSP70 plasma concentrations as determined by ELISA, month 9 reported. The result is expressed as optical density (OD) units. The greater the number; the more autoantibody.

Absolute Survival Percentageduring 9 months of observation

Absolute survival in the two arms calculated by actuarial methods (Kaplan-Meier). These result in percent survival denoted as means and +/- standard error (these are detailed in the outcome table below).

Number of Adverse Events (AE)during the 9 months of observation

AE in the two treatment arms will be compared. Serious adverse events, as defined by national toxicity scale.

Transplant-Free Survivalduring 9 months of observation

Actuarial transplant-free survival in the two arms, calculated by actuarial methods (Kaplan-Meier).

These result in percent survival denoted as means and +/- standard error (these are detailed in the outcome table below).

Hospitalizationsduring 9 months of observation

The number of hospitalizations in the two arms will be compared.

Trial Locations

Locations (7)

University of Minnestoa

🇺🇸

Minneapolis, Minnesota, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Geisinger Medical Center

🇺🇸

Danville, Pennsylvania, United States

Temple University

🇺🇸

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

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