Stem Cell Transplant for Immunodeficiency
(BOLT+BMT Trial)

Recruiting in Palo Alto (17 mi)

Dr. Paul Szabolcs, MD - Pittsburgh, PA ...

Overseen byPaul Szabolcs, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Paul Szabolcs

No Placebo Group

Trial Summary

What is the purpose of this trial?

This study is evaluating whether a lung transplant followed by a stem cell transplant is safe and effective for people with primary immunodeficiency.

Research Team

Paul Szabolcs, MD

Principal Investigator

Division of BMT and Cellular Therapy, Children's Hospital of Pittsburgh of UPMC

Eligibility Criteria

This trial is for people aged 5-45 with primary immunodeficiency and severe lung disease needing a lung transplant. They must have normal liver function, heart function, kidney function, not be pregnant or HIV positive, and agree to birth control post-transplant.

Inclusion Criteria

My kidney function is good.

Your liver enzymes (AST, ALT) and bilirubin levels are within a certain range, and your blood clotting time is normal.

I am between 5 and 45 years old.

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Exclusion Criteria

I do not have an uncontrolled lung infection.

Past or current medical problems or findings from physical examination or laboratory testing that 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.

You have tested positive for HIV or HTLV.

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Treatment Details

Interventions

CD3/CD19 negative allogeneic hematopoietic stem cells (Hematopoietic Stem Cell Transplantation)

Trial OverviewThe study tests if getting new lungs (BOLT) followed by stem cell transplant from a partially-matched donor is safe/effective for immune deficiency diseases. Participants receive CD3/CD19 negative allogeneic hematopoietic stem cells.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: BOLT+BMTExperimental Treatment1 Intervention

All patients will receive a double lung transplant followed by a hematopoietic stem cell transplant. The lungs and stem cells are from the same partially HLA-matched cadaveric donor. Prior to transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device.