pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

June 25, 2024

Primary Completion Date

March 31, 2027

Study Completion Date

March 31, 2027

Conditions
p47Autosomal RecessiveChronic Granulomatous Disease
Interventions
BIOLOGICAL

Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47

"Cryopreserved autologous CD34+ cells transduced ex vivo with the pCCLCHIM-p47 vector containing the human p47phox (NCF1) gene in final formulation and container closure system, ready for intended medical use.~The minimum cell dose for infusion is 3 x 10\^6 CD34+/kg."

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH