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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Study ID
NCT07113743
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Chronic Granulomatous Disease (CGD)

Eligibility Criteria

Sex
ALL
Age
3 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • Busulfan — DRUG
    Conditioning drug
  • Tocilizumab — DRUG
    Monoclonal Antibody
  • Eltrombopag — DRUG
    Thrombopoietin Receptor Agonist
  • Sirolimus — DRUG
    Post transplant immunosuppressant drug
  • pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene — OTHER
    Intervention Infusion on Day 0

Study Details

Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly. Researchers want to see if a lentiviral gene transfer treatment will have the ability to make the patient s immune system more normal, in particular reduce the risk of CGD related infections. The gene transfer takes a person s own stem cells, cultures them to put the normal gene in, then gives the cells back to the person. Objective: To test a gene transfer treatment for X-CGD. Eligibility: Participants aged 3-60 with X-CGD Design: Participants will be screened under protocol 05-I-0123. They will undergo: Medical history Physical exam Heart tests Imaging tests, as needed Blood tests Lung function tests, as needed Dental and audiology exams, if needed Quality of life questionnaire Bone marrow aspiration. A needle will be inserted into the hip bone or breastbone to collect bone marrow. Some screening tests will be repeated during the study. Participants will have an apheresis procedure under protocol 94-I-0073. Stem cells will be collected. Participants will get a series of drugs to prepare them for the gene transfer. Participants will stay at the NIH Clinical Center for a little over a month. They will get a central line. It is a large intravenous (IV) catheter that is placed into a vein of the neck, chest, or arm. They will get chemotherapy and their corrected stem cells through their IV line. Participants will have 12 follow-up outpatient visits in the 2 years after their gene transfer, as well as visits with their local doctor. Then they will enroll in another study for long-term follow-up visits that will last for 13 years.

Key Dates

Start date
Sep 10, 2025
Status verified
Apr 2026
Primary completion
Sep 1, 2028
Completion
Sep 1, 2029

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: X-linked CGD
    Non-randomized single arm

Primary Outcome Measure

Safety [ Time Frame: Throughout the study ]

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892-

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National Institutes of Health Clinical Center· Bethesda, MD

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