Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-60
SponsorPaul Szabolcs
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Pulmonary Fibrosis
Disqualifiers
Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
Patients who have underlying malignant conditions.
Patients who have non-malignant conditions not requiring BMT.
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.
Trial design
Design model
Single group
Treatments tested in this trial
CD3/CD19 negative hematopoietic stem cells
Biological/VaccineNegative selection for CD3/CD19 will be performed on CliniMACS® depletion device and given at time no less than 8 weeks post lung transplantation
Rituximab
DrugTransplantation Conditioning
Alemtuzumab
DrugTransplantation Conditioning
Fludarabine
DrugTransplantation Conditioning
Thiotepa
DrugTransplantation Conditioning
G-CSF
DrugTransplantation conditioning
Hydroxyurea
DrugTransplantation Conditioning
Treatment groups
8 Participants
are divided into 1 treatment groupGroup A: Lung and Bone Marrow TransplantationExperimental treatment 7 interventions
Trial outcomes
Primary outcomes
1
Death
How many, if any, patients die
Time frame
Up to 2 years post stem cell transplant2
Engraftment failure
How many, if any, develop engraftment failure
Time frame
Up to 2 years post stem cell transplant3
Non-hematologic events
Any Grade 4 event that happens at any time points
Time frame
Up to 2 years post stem cell transplant4
Hematological events
Any Grade 4 hematological events
Time frame
after 30 days post stem cell transplantSecondary outcomes
1
Feasibility of patients able to proceed to BMT within 6 months following lung transplantation
The number of patients who are able to proceed to BMT within 6 months following lung transplantation
Time frame
Up to 2 years post stem cell transplant2
Independence
The number of patients who are able to be independent from transfusions and growth factors for at least 7 days
Time frame
up to 2 years post stem cell transplant3
Independence
The number of patients who are able to be independent from transfusions and growth factors for at least 1 month
Time frame
Up to 2 years post stem cell transplant4
Tolerance development to both host and pulmonary grafting
Development of tolerance to both the host and pulmonary graft
Time frame
Up to 2 years post stem cell transplantOther outcomes
1
Pace of immune reconstitution
The pace of immune reconstitution, systemically and in mucosal surfaces
Time frame
Up to 2 years post stem cell transplant2
Mixed chimerism
The number of patients who have the incidence of mixed chimerism (.5% host cells)
Time frame
at Months 1, 3, 6 and 12 post stem cell transplant3
In vitro immune tolerance
The number of patients who have in vitro immune tolerance
Time frame
Up to 2 years post stem cell transplantSponsors and contacts
Click on the lead sponsor to view all of their trials.
Paul Szabolcs
Lead sponsor
University of Pittsburgh
Sponsor institution
Source ClinicalTrials.gov