Gene Therapy for Chronic Granulomatous Disease

Recruiting in Palo Alto (17 mi)

+2 other locations

Overseen byCaroline Y. Kuo, MD

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: University of California, Los Angeles

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial involves taking a patient's own stem cells, correcting a genetic defect in a lab, and then reintroducing these modified cells back into the patient. It targets individuals with Chronic Granulomatous Disease (CGD), a condition where white blood cells can't effectively fight infections. The treatment uses a method to insert a healthy gene into the patient's stem cells, enabling them to produce functional white blood cells. Gene therapy has shown promise in treating Chronic Granulomatous Disease (CGD) by using corrected stem cells.

Eligibility Criteria

This trial is for male patients over 23 months old with X-linked Chronic Granulomatous Disease (CGD), confirmed by DNA tests and lab evidence. They must have had severe infections despite treatment, no perfect bone marrow donor match, and be free of certain viruses like HIV or hepatitis. Patients need to consent to the study's procedures.

Inclusion Criteria

I am a male over 23 months old with confirmed X-CGD and very low NADPH-oxidase activity.

I do not have HIV, hepatitis B or C, adenovirus, parvovirus B19, toxoplasmosis, or an active CMV infection.

I have signed the consent form, or if under 18, I have given my assent.

+3 more

Exclusion Criteria

I haven't received gamma-interferon within the last 30 days.

I cannot undergo leukapheresis or bone marrow harvest due to anemia, heart issues, or severe bleeding disorders.

Contraindication for administration of conditioning medication (Known sensitivity to Busulfan)

+11 more

Participant Groups

The trial is testing a new gene therapy using a lentiviral vector called G1XCGD in CGD patients. It involves taking the patient's own bone marrow cells, modifying them with this vector to correct the genetic defect, then returning them back into the patient after chemotherapy conditioning.

2Treatment groups

Experimental Treatment

Group I: Lentiviral G1XCGD Gene Therapy, Part BExperimental Treatment1 Intervention

Transplantation with autologous CD34+ stem cells corrected with X1XCGD lentiviral vector after modified myeloreductive conditioning including increased monitoring and rescue treatment

Group II: Lentiviral G1XCGD Gene Therapy, Part AExperimental Treatment1 Intervention

Transplantation with autologous CD34+ stem cells corrected with X1XCGD lentiviral vector after myeloreductive conditioning