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Double-blind Placebo-controlled Pilot Study of Sirolimus in Idiopathic Pulmonary Fibrosis (IPF)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01462006
Recruitment Status : Completed
First Posted : October 28, 2011
Last Update Posted : March 22, 2021
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Borna Mehrad, MD, University of Virginia

Brief Summary:
Idiopathic pulmonary fibrosis (IPF) is an illness characterized by progressive decline in lung function and premature death from respiratory failure. Fibrocytes are a novel population of bone marrow-derived circulating progenitor cells that have been shown to traffic to the lungs and contribute to fibrosis in animal models of pulmonary fibrosis, and whose numbers correlate with the degree of fibrosis and with survival in human pulmonary fibrosis. The investigators propose to test the hypothesis that therapy with the mTOR inhibitor, sirolimus, reduces the number of circulating fibrocytes in patients with IPF. The investigators propose to test this hypothesis in short-term pilot trial of sirolimus in patients with IPF to determine its effect on the number and phenotype of circulating fibrocytes.

Condition or disease Intervention/treatment Phase
Idiopathic Pulmonary Fibrosis Diffuse Parenchymal Lung Disease Interstitial Lung Disease Drug: sirolimus Other: Placebo Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-blind Placebo-controlled Pilot Study of Sirolimus in IPF
Actual Study Start Date : October 2011
Actual Primary Completion Date : March 2016
Actual Study Completion Date : March 2016

Arm Intervention/treatment
Experimental: Sirolimus Drug: sirolimus
randomized to drug or placebo, followed by washout, followed by crossover

Placebo Comparator: Placebo Other: Placebo
randomized to drug or placebo, followed by washout, followed by crossover

Primary Outcome Measures :
  1. fibrocytes [ Time Frame: up to 22 weeks ]
    change in peripheral blood concentration of CXCR4+ fibrocytes

  2. number of subjects with drug side-effects [ Time Frame: up to 22 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   21 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male and female patients 21-85 years of age
  2. Individuals diagnosed with IPF, based on:

    • clinical symptoms consistent with idiopathic pulmonary fibrosis (IPF) of > 3 months duration, plus
    • histologically diagnosed UIP or diagnostic chest high resolution CT features of UIP, plus
    • negative workup for known causes of UIP
  3. Ability to understand a written informed consent form and comply with the requirements of the study.

Exclusion Criteria:

  1. Clinical features or known diagnosis of an active infection, including untreated latent tuberculosis
  2. Clinical features or known diagnosis of malignancy
  3. Known diagnosis of an interstitial lung disease other than IPF including but not limited to sarcoidosis, hypersensitivity pneumonitis, non-specific interstitial pneumonia (NSIP).
  4. History of clinically significant environmental exposures known to cause interstitial lung disease (including but not limited to drugs, asbestos, silica, beryllium, radiation, domestic birds, etc).
  5. Diagnosis of any connective tissue disease (including but not limited to scleroderma, SLE, rheumatoid arthritis) or vasculitides according to the American College of Rheumatology criteria.
  6. Systolic blood pressure < 100 or >145 mm Hg or diastolic blood pressure < 50 or >90 mmHg
  7. Evidence of active infection within 1 week prior to enrollment.
  8. Recently started (<8 weeks prior to baseline visit) or planned cardiopulmonary rehabilitation program before conclusion of the study
  9. History of unstable or deteriorating cardiac disease, including but not limited to: myocardial infarction, coronary artery bypass surgery or angioplasty within the past 6 months, congestive heart failure requiring hospitalization within the past 6 months, or uncontrolled arrhythmia
  10. History of unstable or deteriorating neurologic disease, including but not limited to: TIAs or stroke
  11. Pregnant or lactating females. Females of child bearing potential are required to have a negative serum or urine pregnancy test prior to treatment and agree to practice abstinence or prevent pregnancy by at least a barrier method of birth control.
  12. Liver panel above specific limits at screening: Total bilirubin >1.5-fold upper limit of normal, AST, ALT or alkaline phosphatase > 3-fold upper limit of normal at screening.
  13. Hematology outside of specified limits, WBC <2,500/ mm3, hematocrit <30, platelets <100,000/mm3 at screening.
  14. Investigational therapy for any indication within 28 days prior to treatment.
  15. Current treatment with drugs that are strong inhibitors of CYP3A4 or P-gp, namely bromocriptine, cimetidine, cisapride, clotrimazole, danazol, diltiazem, fluconazole, HIV-protease inhibitors (e.g., ritonavir, indinavir), metoclopramide, nicardipine, troleandomycin, verapamil
  16. Inability or unwillingness to comply with the requirements for the trial.

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): NCT01462006

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United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Sponsors and Collaborators
University of Virginia
National Heart, Lung, and Blood Institute (NHLBI)
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Principal Investigator: Borna Mehrad, MD University of Florida
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Responsible Party: Borna Mehrad, MD, Professor, Department of Medicine, Pulmonary and Critical Care, University of Virginia
ClinicalTrials.gov Identifier: NCT01462006    
Other Study ID Numbers: 15282
R01HL098329 ( U.S. NIH Grant/Contract )
First Posted: October 28, 2011    Key Record Dates
Last Update Posted: March 22, 2021
Last Verified: March 2021
Additional relevant MeSH terms:
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Lung Diseases
Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Lung Diseases, Interstitial
Pathologic Processes
Respiratory Tract Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs