*Immunodeficiency and End-Stage Lung Disease Treatment with BOLT+BMT Procedure – Phase I/II

Protocol Description

This study seeks to determine whether bilateral orthotopic lung transplantation (BOLT) followed by partially matched hematopoietic stem cell transplantation (HSCT) from the same donor is safe and effective for patients with primary immunodeficiency (PID) and end-stage lung disease. Although HSCT is a curative, life-saving treatment for patients with PID, these patients are often ineligible for HSCT because of pulmonary complications brought on by chronic or recurrent infections resulting from the PID. Researchers believe that by restoring pulmonary function with a lung transplant, patients may be able to proceed to HSCT to cure the underlying immunodeficiency and, quite possibly, provide freedom from all immunosuppressive drugs.

Study Level: Phase I/II

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting participants of both genders between the ages of 5 and 45 who have evidence of an underlying primary immunodeficiency for which HSCT is clinically indicated and who concurrently have end-stage lung disease.
Males: 5 to 45 years old
Females: 5 to 45 years old

Requirements

Participants in this study will have both a double lung transplant and a stem cell transplant. The lung transplant will occur at least 6 weeks before the stem cell transplant, allowing sufficient time for recovery from the lung transplant. Due to the severity of participants’ lung disease, the process includes a reduced intensity regimen of the chemotherapy, radiation and drugs that are needed suppress the immune system so the body does not reject transplanted stem cells.
Visits: Requires hospitalization and surgery 
Duration: Initially after discharge, patients will be seen at the bone marrow transplant clinic several times a week, then at 6 months, 1 and 2 years.

Status: Open for Enrollment

Additional Information

Study Description from the National Institutes of Health

Primary Investigator

Paul Szabolcs, MD

Contact Information

For information about the study or enrollment, please contact:
Shawna McIntyre, Clinical Research Supervisor
412-692-5552