Safety and Efficacy of PMT Therapy of hPAP

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

June 26, 2023

Primary Completion Date

October 1, 2025

Study Completion Date

October 1, 2038

Conditions
Hereditary Pulmonary Alveolar Proteinosis
Interventions
COMBINATION_PRODUCT

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.

Trial Locations (1)

45229

RECRUITING

Cincinnati Children's Hospital Medical Center, Cincinnati

All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

University of South Florida

OTHER

lead

Children's Hospital Medical Center, Cincinnati

OTHER