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.
• Subject and/or parent guardian must be able to understand and provide informed consent.
• Male or female, 5 through 45 years old, inclusive, at the time of informed consent.
• 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
• Hyper IgE Syndrome or Job Syndrome
• CD40 or CD40L deficiency
• Wiskott-Aldrich Syndrome
• Mendelian Susceptibility to Mycobacterial Disease [6]
• GATA2 Associated Immunodeficiency NOTE: A genetic diagnosis is recommended, but not required.
• Patients must have evidence of end-stage lung disease and be candidates for bilateral orthotopic lung transplant as determined by the lung transplant team.
• GFR ≥ 50 mL/min/1.73 m2.
• AST, ALT ≤ 4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR.
• Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%.
• Negative pregnancy test for females >10 years old or who have reached menarche, 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 and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting.
This content was sourced from clinicaltrials.gov