Safety and Efficacy of PMT Therapy of hPAP

NCTID	NCT05761899 (View at clinicaltrials.gov)
Description	The major goal of this study is to evaluate a new type of cell transplantation therapy for individuals with hereditary PAP, study a new treatment that may be useful for treatment of other diseases, and research mechanisms that drive the development and function of lung macrophages. (Show More)
Indication	Hereditary Pulmonary Alveolar Proteinosis
Compound Name	Gene-Corrected Macrophages (SIN-LV/EFS/CSF2RA)
Sponsor	Children's Hospital Medical Center, Cincinnati
Funder Type	Other
Status	Recruiting
Enrollment Count	3

Target Gene/Variant	CSF2RA
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Broncoscopy
Drug Product Type	Viral vector
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	11.1 x 10^6 cells/kg ideal body weight
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
Recent Updates	Licensed to Altius therapeutics