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

Summary

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.

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

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID)

  lead NIH

Principal Investigators

• Elizabeth M Kang, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

3 Years

Max Age

60 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-09-10

Primary Completion

2028-09-01

Completion

2029-09-01

FDA Drug

Yes

Countries

• United States

Study Locations