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Continuation of Nintedanib After Single Lung Transplantation in IPF Subjects

Phase 2
Terminated
Conditions
Idiopathic Pulmonary Fibrosis
Lung Transplant; Complications
Interventions
Drug: Placebo Oral Tablet
Registration Number
NCT03562416
Lead Sponsor
Temple University
Brief Summary

The aim of this study is to assess the utility of nintedanib therapy in addition to usual transplant care in single lung transplant recipients with idiopathic pulmonary fibrosis (IPF). The investigators hypothesize that in IPF subjects who undergo single lung transplantation the administration of nintedanib 150 mg twice daily in addition to usual transplant care will result in better preservation of lung function at 24 months.

Detailed Description

Lung transplantation is the only treatment option that augments survival in patients with idiopathic pulmonary fibrosis (IPF). Despite several advancements in lung transplantation over the past three decades, long-term survival rates have remained low compared to other solid organ transplantations. The median survival after lung transplantation is only 5.8 years. Multiple factors account for the relatively low survival post-transplant, but chronic rejection resulting in obliterative bronchiolitis is a predominate cause. Further research is needed to develop medical therapeutic interventions that improve survival in IPF patients who undergo only single lung transplantation.

Nintedanib, a novel tyrosine kinase inhibitor, exhibits antifibrotic properties via multiple mechanisms including the inhibition of the receptor tyrosine kinases platelet derived growth factor (PDGF) receptor, fibroblast growth factor (FGF) receptor, and vascular endothelial growth factor (VEGF) receptor. Several mediators of pulmonary fibrosis including VEGF, FGF, and transforming growth factor beta (TGF-β) have also been implicated in the pathogenesis of bronchiolitis obliterans syndrome (BOS), the most common type of chronic lung allograft rejection.

Nintedanib is safe to continue until the time of lung transplantation and has not been shown to worsen perioperative outcomes in small case series, single center cohorts and our center's personal experience. The current practice in lung transplant medicine is to discontinue antifibrotic therapy after lung transplantation in IPF. In IPF patients who undergo single lung transplant, nintedanib therapy has the potential to preserve lung function in both the native fibrotic lung and the new lung allograft.

The investigators propose a randomized and placebo-controlled single center pilot trial comparing nintedanib therapy plus usual care to usual care only in IPF patients after single lung transplant. The investigators hypothesize that in IPF subjects who undergo single lung transplantation the administration of nintedanib 150 mg twice daily in addition to usual transplant care will result in better preservation of lung function at 24 months.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
1
Inclusion Criteria
  • Adults between the ages of 35-70.
  • Lung transplantation listing diagnosis of pulmonary fibrosis
  • Recipient of single lung transplantation within the past 60 days
Exclusion Criteria
  • History of intolerability to nintedanib (i.e. discontinued nintedanib in the pre-transplant period due to adverse drug effects)

  • Liver transaminase elevation (AST or ALT > 1.5X the upper limit of normal)

  • Total bilirubin > 1.5X the upper limit of normal

  • Drugs that interfere with the metabolism or elimination of nintedanib or its metabolites - St. John's wort, carbamazepine, phenytoin, rifampin, dexamethasone, and others.

  • Any history of bronchial anastomosis dehiscence or stenosis

  • Bleeding risk, defined as any of the following:

    • Full-dose therapeutic anticoagulation (i.e. vitamin K antagonist, direct thrombin inhibitors, etc.)
    • History of hemorrhagic central nervous system (CNS) event within 12 months of enrollment
    • Coagulation parameters: international normalized ratio (INR) > 2, prolongation of prothrombin time (PT) and partial thromboplastin time (PTT) by > 1.5X the upper limit of normal at enrollment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
NintedanibNintedanibNintedanib 150 mg tablet by mouth twice daily for 24 months.
PlaceboPlacebo Oral TabletPlacebo tablet by mouth twice daily for 24 months
Primary Outcome Measures
NameTimeMethod
Change in FVCBaseline to 24 months

Change in forced vital capacity (FVC)

Change in FEV1Baseline to 24 months

Change in forced expiratory volume in 1 second (FEV1)

Secondary Outcome Measures
NameTimeMethod
Bronchial stenosisBaseline to 24 months

Incidence of surgical anastomosis bronchial stenosis

Peripheral blood flow cytometry - CD8 T cellsDay 300

CD8 T cell concentration in peripheral blood (cells/µL)

Vascular endothelial growth factor (VEGF) - serumBaseline to day 300

Change in serum concentration for VEGF (pg/mL)

Bronchiolitis obliterans syndromeBaseline to 24 months

Incidence of bronchiolitis obliterans syndrome (BOS)

Drug discontinuationBaseline to 24 months

Study drug discontinuation rate due to adverse drug event

Fibroblast growth factor (FGF) - BALBaseline to day 300

Change in BAL biomarker concentration for FGF (pg/mL)

Peripheral blood flow cytometry - macrophagesDay 300

Macrophage concentration in peripheral blood (cells/µL)

Adverse drug eventsBaseline to 24 months

Incidence of adverse drug events (i.e. elevation of liver transaminases greater than 3 times the upper limit of normal, diarrhea, nausea, vomiting, anorexia, GERD)

Platelet derived growth factor (PDGF) - serumBaseline to day 300

Change in serum biomarker concentration for PDGF (pg/mL)

Platelet derived growth factor (PDGF) - BALBaseline to day 300

Change in BAL biomarker concentration for PDGF (pg/mL)

Fibroblast growth factor (FGF) - serumBaseline to day 300

Change in serum concentration for FGF (pg/mL)

Peripheral blood flow cytometry - CD4 T cellsDay 300

CD4 T cell concentration in peripheral blood (cells/µL)

Peripheral blood flow cytometry - neutrophilsDay 300

Neutrophil concentration in peripheral blood (cells/µL)

Bronchial dehiscenceBaseline to 24 months

Incidence of surgical anastomosis bronchial stenosis

Acute cellular rejectionBaseline to 24 months

Incidence of acute cellular rejection of lung allograft

Vascular endothelial growth factor (VEGF) - BALBaseline to day 300

Change in BAL concentration for VEGF (pg/mL)

Survivalbaseline to 24 months

Survival and time to death/cause of death (if applicable) of study subjects

Trial Locations

Locations (1)

Temple University Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

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