Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure

  • STATUS
    Recruiting
  • End date
    Dec 16, 2026
  • participants needed
    8
  • sponsor
    Paul Szabolcs
Updated on 16 February 2021
COPD
immunosuppressant
pulmonary disease
emphysema
lung transplant
a hemoglobin

Summary

The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.

Description

The primary purpose of the study is to evaluate the safety and efficacy of performing lung transplantation followed by cadaveric, partially HLA-matched (1/6 HLA-match with an identical ABO blood type) CD3+/CD19+ depleted bone marrow transplantation in bone marrow failure and end-stage lung disease. Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive, and fatal interstitial lung disease for which lung transplantation is the only therapy shown to prolong survival. Given the association of IPF with hematologic cytopenias and bone marrow failure, it is proposed that a tandem lung transplantation and bone marrow transplantation from a single cadaveric donor could be successful. This protocol focuses on performing combined transplantation for candidates that are unable to undergo standard lung transplantation. Lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, and to restore hematologic function post BMT transplantation. The secondary objectives are to evaluate the feasibility and long-term complications associated with combined solid organ and BMT including the ability to initiate and successfully withdraw from immunosuppression following BMT and to attain independence from growth factors, red blood cell or platelet transfusions.

Details
Condition Pulmonary Fibrosis, Pulmonary Fibrosis, Idiopathic Pulmonary Fibrosis, Idiopathic Pulmonary Fibrosis, Emphysema or COPD, usual interstitial pneumonia
Treatment Rituximab, G-CSF, hydroxyurea, Fludarabine, alemtuzumab, thiotepa, CD3/CD19 negative hematopoietic stem cells
Clinical Study IdentifierNCT03500731
SponsorPaul Szabolcs
Last Modified on16 February 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Individuals must meet all of the following criteria in order to be eligible
for this study
Subject must be able to understand and provide informed consent
Male or female, 18 through 60 years old, inclusive, at the time of informed consent
Meet criteria for UNOS listing for lung transplantation
Patients must have evidence of end stage lung disease. Examples of such diseases include but are not limited to
Pulmonary Fibrosis
COPD/Emphysema
Patients must have evidence of bone marrow failure with abnormal low cell count in at least one hematopoietic line, making the patient a poor candidate for long-term immunosuppressive therapy. Eligible patients must meet at least one of the following
criteria
Unexplained, non-drug induced neutropenia with absolute neutrophils counts of <1500/L the previous year, confirmed by repeat testing
Unexplained, non-drug induced thrombocytopenia with mean platelets counts of <100,000/L the previous year, confirmed by repeat testing
Unexplained, non-hemolytic anemia, with a hemoglobin level of < 12 g/dL the previous year, confirmed by repeat testing
GFR 45 mL/min/1.73 m2
AST, ALT 4x upper limit of normal, total bilirubin 2.5 mg/dL, normal INR, albumin >3.0 g/dL
Cardiac ejection fraction 40% or shortening fraction 26%
Negative pregnancy test for females, unless surgically sterilized
All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defect
Subject will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting

Exclusion Criteria

Individuals who meet any of these criteria are not eligible for this study
Inability or unwillingness of a participant to give written informed consent or comply with study protocol
Patients who have underlying malignant conditions
HIV positive by serology or PCR, HTLV positive by serology
Females who are pregnant or who are lactating
Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product
Uncontrolled infection, as determined by the appropriate imaging and/or confirmatory testing e.g. blood cultures, PCR testing, etc
Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of transplant
Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study
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