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

Who is this study for? Patients with combined lung and bone marrow failure
What treatments are being studied? CD3/CD19 negative hematopoietic stem cells+Rituximab+Alemtuzumab+Fludarabine+Thiotepa+G-CSF+Hydroxyurea
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug, Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 60
Healthy Volunteers: f
View:

∙ 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/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

• 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.

Locations
United States
Pennsylvania
Children's Hospital of Pittsburgh of UPMC
RECRUITING
Pittsburgh
UPMC Presbyterian
RECRUITING
Pittsburgh
Contact Information
Primary
Paul Szabolcs, M.D.
Paul.Szabolcs@chp.edu
412-692-5427
Backup
Shawna H McIntyre, RN
mcintyresm@upmc.edu
412-692-5552
Time Frame
Start Date: 2018-04-19
Estimated Completion Date: 2026-12
Participants
Target number of participants: 8
Treatments
Experimental: Lung and Bone Marrow Transplantation
All patients will undergo a cadaveric, partially HLA-matched lung transplantation followed by a CD3+/CD19+ depleted BMT from the same donor. In this study, the investigators will use a ≥1/6 HLA-matched T cell depleted bone marrow transplantation from a cadaveric organ donor with an identical ABO blood type as the recipient. Prior to transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device.~Subjects will undergo lung transplantation utilizing standard induction regimens selected by the CO-PIs based on the subject's underlying comorbidities and allosensitization. Rituximab may be initiated prior to the lung transplantation with tacrolimus as the ongoing maintenance immunosuppression.~Subjects will undergo BMT utilizing CD3+/CD19+-depleted bone marrow with bone marrow conditioning beginning no less than 8 weeks after lung transplantation. Bone marrow will be recovered alongside solid organs and will be processed and cryopreserved.
Sponsors
Leads: Paul Szabolcs

This content was sourced from clinicaltrials.gov