3
Participants
Start Date
June 26, 2023
Primary Completion Date
October 1, 2025
Study Completion Date
October 1, 2038
Gene-Corrected Macrophages administered by bronchoscopic instillation
This study will evaluate administration of autologous bone marrow CD34+ cell-derived, CSF2RA lentiviral vector-transduced macrophages (CSF2RA gene-corrected macrophages) by bronchoscopic instillation into individual lung segments on three occasions at 2-month intervals in patients with hPAP. The target (maximum) number of cells to be administered is 778 million gene-corrected macrophages per 70 kg patient, which is equal to 11.1 million cells/kg of ideal body weight.
RECRUITING
Cincinnati Children's Hospital Medical Center, Cincinnati
National Heart, Lung, and Blood Institute (NHLBI)
NIH
University of South Florida
OTHER
Children's Hospital Medical Center, Cincinnati
OTHER