NCT05207657View on ClinicalTrials.gov

Lentiviral Gene Therapy for p47 AR-CGD

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

March 20, 2023

Primary Completion Date

April 1, 2029

Study Completion Date

April 1, 2029

Conditions
P47-Phox, Deficiency of
Interventions
DRUG

Lentiviral vector transduced CD34+ cells

Gene therapy for p47 AR-CGD will be performed by introduction a normal copy of the NCF-1 gene into the blood forming stem cells (CD34+ cells) of the patient's bone marrow by using a type of gene delivery system (in this trial called a lentiviral vector). The gene corrected cells are then transplanted back into the patient

Trial Locations (1)

WC1N 3JH

RECRUITING

Great Ormond Street Hospital, London

All Listed Sponsors
lead

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER