Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure

This study is exploring a new approach for people with severe lung diseases like Idiopathic Pulmonary Fibrosis (IPF), emphysema, or COPD, who also have bone marrow failure. It tests whether having a lung transplant first, followed by a bone marrow transplant, can help restore both lung and blood cell function. The study uses specific treatments like Rituximab, Alemtuzumab, Fludarabine, and Thiotepa for conditioning, and then gives specially prepared hematopoietic stem cells (blood-forming cells). Researchers are looking at safety, including death, transplant failure, and other medical problems, for up to two years after the stem cell transplant. You might be able to join if you are between 18 and 60 years old and meet criteria for a lung transplant due to end-stage lung disease. The study is currently unclear on its recruitment status and plans to enroll 8 participants.

Study design
This is an interventional study with no specified phase, designed to enroll 8 participants. It does not mention randomization or blinding.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 2 years after the stem cell transplant to monitor for death, engraftment failure, and non-hematologic events.

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NCT03500731

Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure

Recruiting
PHASE1Ages 18–60InterventionalTreatment
Paul Szabolcs
~8 participants
Updated 2026-01-13 on ClinicalTrials.gov
What's tested:CD3/CD19 negative hematopoietic stem cellsRituximabAlemtuzumabFludarabineThiotepaG-CSF

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Death
Measured over Up to 2 years post stem cell transplant
+7 more outcomes measured
Idiopathic Pulmonary Fibrosis
Emphysema or COPD
2 sites across 1 states
Pennsylvania2

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Eligibility criteria

Inclusion

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.
  • DeathUp to 2 years post stem cell transplant

    How many, if any, patients die

  • Engraftment failureUp to 2 years post stem cell transplant

    How many, if any, develop engraftment failure

  • Non-hematologic eventsUp to 2 years post stem cell transplant

    Any Grade 4 event that happens at any time points

  • Hematological eventsafter 30 days post stem cell transplant

    Any Grade 4 hematological events

  • BOS Scoreat 1 year post lung transplant

    Bronchiolitis Obliterans Syndrome (BOS) score based off patient pulmonary function testing. Graded on scale (BOS0 to BOS3), BOS0 having a better outcome then BOS3

  • T-cell Chimerismat 12 months post stem cell transplant

    The number of patients who have ≥25% donor T-cell chimerism

  • Myeloid chimerismat 12 months post stem cell transplant

    The number of patients with myeloid disorders who attain ≥ 10% myeloid chimerism

  • Restoration of blood cell count (in absence of growth factors)at 12 months post stem cell transplant

    Absolute neutrophil count (ANC)≥1000 per microliter of blood, platelets ≥50000 per microliter of blood and hematocrit ≥8 grams per deciliter of blood