RECRUITING

Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure

Conditions

Idiopathic Pulmonary Fibrosis Emphysema or COPD Lung Transplantation Stem Cell Transplantation Bone marrow transplantation Cadaveric donor Unrelated donor HLA-Mismatch BMT HSCT IPF Pulmonary fibrosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Lung Transplant in Tandem With Bone Marrow Transplant for Combined Lung and Bone Marrow Failure

Quick Facts

Study Start:2018-04-19

Study Completion:2026-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT03500731

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 60 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT

Inclusion Criteria	Exclusion Criteria
1. Subject must be able to understand and provide informed consent. 2. Male or female, 18 through 60 years old, inclusive, at the time of informed consent. 3. Meet criteria for UNOS listing for lung transplantation. 4. Patients must have evidence of end stage lung disease. Examples of such diseases include but are not limited to: * Pulmonary Fibrosis * COPD/Emphysema 5. 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/µL the previous year, confirmed by repeat testing * Unexplained, non-drug induced thrombocytopenia with mean platelets counts of \<100,000/µL 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 6. GFR ≥45 mL/min/1.73 m2. 7. AST, ALT ≤4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR, albumin \>3.0 g/dL 8. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%. 9. Negative pregnancy test for females, unless surgically sterilized. 10. 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. 11. Subject will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting.	1. Inability or unwillingness of a participant to give written informed consent or comply with study protocol. 2. Patients who have underlying malignant conditions. 3. Patients who have non-malignant conditions not requiring BMT. 4. HIV positive by serology or PCR, HTLV positive by serology. If HTLV serology is positive, it will be confirmed by nucleic acid testing (NAT). If HTLV NAT is negative, subject will remain eligible regardless of HTLV serology result. 5. Females who are pregnant or who are lactating. 6. Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product. 7. Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration. NOTE: Pulmonary colonization with multiple organisms is common and will not be considered an exclusion criterion. 8. Uncontrolled infection, as determined by the appropriate imaging and/or confirmatory testing e.g. blood cultures, PCR testing, etc. 9. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of transplant. 10. 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.

Inclusion Criteria

Exclusion Criteria

1. Subject must be able to understand and provide informed consent.
2. Male or female, 18 through 60 years old, inclusive, at the time of informed consent.
3. Meet criteria for UNOS listing for lung transplantation.
4. Patients must have evidence of end stage lung disease. Examples of such diseases include but are not limited to:
* Pulmonary Fibrosis
* COPD/Emphysema
5. 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/µL the previous year, confirmed by repeat testing
* Unexplained, non-drug induced thrombocytopenia with mean platelets counts of \<100,000/µL 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
6. GFR ≥45 mL/min/1.73 m2.
7. AST, ALT ≤4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR, albumin \>3.0 g/dL
8. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%.
9. Negative pregnancy test for females, unless surgically sterilized.
10. 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.
11. Subject will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting.

1. Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
2. Patients who have underlying malignant conditions.
3. Patients who have non-malignant conditions not requiring BMT.
4. HIV positive by serology or PCR, HTLV positive by serology. If HTLV serology is positive, it will be confirmed by nucleic acid testing (NAT). If HTLV NAT is negative, subject will remain eligible regardless of HTLV serology result.
5. Females who are pregnant or who are lactating.
6. Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product.
7. Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration. NOTE: Pulmonary colonization with multiple organisms is common and will not be considered an exclusion criterion.
8. Uncontrolled infection, as determined by the appropriate imaging and/or confirmatory testing e.g. blood cultures, PCR testing, etc.
9. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of transplant.
10. 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.

Contacts and Locations

Study Contact

Paul Szabolcs, M.D.

CONTACT

412-692-5427

Paul.Szabolcs@chp.edu

Shawna H McIntyre, RN

CONTACT

412-692-5552

mcintyresm@upmc.edu

Study Locations (Sites)

UPMC Presbyterian

Pittsburgh, Pennsylvania, 15214

United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224

United States

Collaborators and Investigators

Sponsor: Paul Szabolcs

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-04-19

Study Completion Date2026-12

Study Record Updates

Study Start Date2018-04-19

Study Completion Date2026-12

Terms related to this study

Keywords Provided by Researchers

Lung Transplantation
Stem Cell Transplantation
Idiopathic Pulmonary Fibrosis
Emphysema or COPD
Bone marrow transplantation
Cadaveric donor
Unrelated donor
HLA-Mismatch
BMT
HSCT
IPF
Pulmonary fibrosis

Additional Relevant MeSH Terms

Idiopathic Pulmonary Fibrosis
Emphysema or COPD