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

Enrolling by invitation · Phase 1/Phase 2

Conditions studied: Chronic Granulomatous Disease (CGD)

In brief

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 facts

Study ID

NCT07113743

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

10

Starts

2025-09-10

Expected to finish

2029-09-01

Last updated by the study team

2026-07-24

Who can join

Age: 3 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• An individual who meets any of the following criteria will be excluded from participation in this study:
• Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements.
• Contraindication for leukapheresis (anemia Hb <8 g/dl, cardiovascular instability, severe coagulopathy).
• Patients who are unable to lie prone during the bone marrow harvesting procedure (in the case of bone marrow harvest, contraindication to general anesthesia).
• Have a 10/10 HLA identical (A,B,C,DR,DQ) family or unrelated adult donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure.
• Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies.
• Altered organ function as outlined below observed within 8 weeks of entering this trial.
• a. Hematologic
• i. Anemia (hemoglobin < 8 g/dl).
• ii. Neutropenia (absolute granulocyte count <1,000/mm3 ).
• iii. Thrombocytopenia (platelet count < 150,000/mm3).
• iv. Prothrombin Time (PT) INR or Partial thromboplastin time (PTT) > 2 X the upper limits of normal (ULN) (patients with a correctable deficiency controlled on medication will not be excluded).
• v. Cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow.
• b. Infectious
• i. Evidence of infection with HIV-1 and -2, Hepatitis B, Hepatitis C, adenovirus, parvovirus B 19 or toxoplasmosis within 8 weeks prior to mobilization/apheresis or bone marrow harvest. Cytomegalovirus (CMV) infection is allowable as long as the infection is under control.
• ii. History of infection with mycobacteria or Bacille Calmette-Guerin (BCG) vaccination.
• c. Pulmonary
• i. Resting O2 saturation by pulse oximetry < 90% on room air.
• d. Cardiac
• i. Abnormal electrocardiogram (ECG) indicating cardiac pathology.
• ii. Uncorrected congenital cardiac malformation with clinical symptomatology.
• iii. Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, hypotension.
• iv. Poor cardiac function as evidenced by LV ejection fraction <40% on echocardiogram.
• e. Neurological
• i. Significant neurologic abnormality by examination.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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