Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

September 10, 2025

Primary Completion Date

September 1, 2028

Study Completion Date

September 1, 2029

Conditions
Chronic Granulomatous Disease (CGD)
Interventions
DRUG

Busulfan

Conditioning drug

DRUG

Tocilizumab

Monoclonal Antibody

DRUG

Eltrombopag

Thrombopoietin Receptor Agonist

DRUG

Sirolimus

Post transplant immunosuppressant drug

OTHER

pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene

Intervention Infusion on Day 0

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH