Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
Lung Transplant in Tandem With Bone Marrow Transplant for Combined Lung and Bone Marrow Failure
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 60 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests whether a lung transplant before a bone marrow transplant can improve lung function enough to allow the second transplant. The bone marrow transplant aims to restore blood cell production. It is for adults ages 18 to 60 with severe lung disease and bone marrow failure.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be 18 to 60 years old, understand the study, and give informed consent.
- Meet UNOS rules for the lung transplant waiting list and have end-stage lung disease, such as lung scarring or COPD.
- Have bone marrow failure with low blood cell counts that make long-term treatment with immune-weakening drugs a poor choice.
- Have at least one unexplained low blood count in the past year, confirmed by repeat tests: neutrophils (infection-fighting cells) below 1,500/µL, average platelets (clotting cells) below 100,000/µL, or hemoglobin (the oxygen-carrying protein) below 12 g/dL. Low neutrophils or platelets must not be caused by drugs; low hemoglobin must not be caused by red blood cell breakdown.
- Meet kidney and liver test limits: kidney filtering rate at least 45; liver enzymes AST and ALT no more than 4 times normal; bilirubin no more than 2.5 mg/dL; normal INR blood-clotting test; and albumin above 3.0 g/dL.
- Meet heart pumping limits: ejection fraction at least 40% or shortening fraction at least 26%. These tests measure how well the heart pumps.
- Women must have a negative pregnancy test unless surgically sterilized. Women who can become pregnant and sexually active men must agree to use FDA-approved birth control for up to 24 months after the bone marrow transplant, or as long as they take medicines that could harm a pregnancy or baby.
- Receive counseling about the risk of losing the ability to have children after the bone marrow transplant, and advice about saving sperm or collecting eggs.
Who can't join
- Cannot or will not give written informed consent or follow study rules.
- Have cancer, or have a non-cancer condition that does not need a bone marrow transplant.
- Test positive for HIV or have confirmed HTLV infection. A positive HTLV screening test does not rule you out if the follow-up NAT test is negative.
- Are pregnant or breastfeeding.
- Are allergic to DMSO or another ingredient used to make the stem cell product.
- Have an uncontrolled infection, including a lung infection. Germs living in the lungs alone do not rule you out.
- Received a live, weakened vaccine within 4 weeks before transplant.
- Have other health problems or test findings that the study doctor believes could increase risk, prevent you from following study rules, or affect the study results.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: Individuals must meet all of the following criteria in order to be eligible for this study. 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. Exclusion Criteria: Individuals who meet any of these criteria are not eligible for this study. 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.
Study sites (2)
PA
- UPMC Presbyterian · Pittsburgh, PA 15214John McDyer, MD · Study contact · 412-648-6161 · mcdyerjf@upmc.eduMore trials in Pittsburgh
- Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA 15224Shawna H McIntyre, RN · Study contact · 412-692-5552 · mcintyresm@upmc.eduPaul Szabolcs, MD · Principal investigatorMore trials in Pittsburgh