Paul Szabolcs
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.
This is an original IND for an investigator initiated phase I/II study. The primary purpose of the study is to evaluate the safety and efficacy of performing bilateral orthotopic lung transplantation (BOLT) followed by cadaveric, partially HLA-matched CD3+/CD19+-depleted hematopoietic stem cell transplantation (HSCT) from the same donor for patients with primary immunodeficiency diseases (PID) and end-stage lung disease. For many patients with primary immunodeficiencies, HSCT is a curative, life-saving therapy, resulting in restoration of function in the immune system. Patients with primary im…
Inclusion Criteria 1. Subject and/or parent guardian must be able to understand and provide informed consent. 2. Male or female, 5 through 45 years old, inclusive, at the time of informed consent. 3. Patients must have evidence of an underlying primary immunodeficiency for which BMT is clinically indicated. Examples of such diseases include, but are not limited to: * Severe Combined Immunodeficiency * Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome * Severe Chronic Neutropenia * Chronic Granulomatous Disease…
Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection and given at time no less than 8 weeks post lung transplant.
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