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Gene Therapy

Gene Therapy for Chronic Granulomatous Disease

Phase 1 & 2
Recruiting
Led By Caroline Y. Kuo, MD
Research Sponsored by University of California, Los Angeles
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male X-CGD patients > 23 months of age with molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 95% of the biochemical activity of the NADPH-oxidase
Must not have co-infection with Human Immunodeficiency Virus (HIV)-1 or -2, hepatitis B virus or hepatitis C virus, adenovirus, parvovirus B 19, toxoplasmosis, or active infection with CMV
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years
Awards & highlights

Study Summary

This trial is testing a new gene therapy that may help people with an immunodeficiency disorder that prevents white blood cells from effectively killing bacteria, fungi and other microorganisms.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects may include reactions related to bone marrow harvest and infusion such as pain or bleeding, complications from chemotherapy like nausea or hair loss, and risks associated with gene therapy which could range from mild immune responses to more serious conditions affecting blood cell growth.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a male over 23 months old with confirmed X-CGD and very low NADPH-oxidase activity.
Select...
I do not have HIV, hepatitis B or C, adenovirus, parvovirus B19, toxoplasmosis, or an active CMV infection.
Select...
I don't have a perfect match donor in the national registries.
Select...
I have had a severe infection or inflammation that needed hospital care despite treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Granulocyte
The incidence of adverse events assessed by CTCAE v4
Other outcome measures
Immunophenotyping
Concentration of gp91 protein produced in response to the corrected gene

Trial Design

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

Find a Location

Who is running the clinical trial?

University of California, Los AngelesLead Sponsor
1,525 Previous Clinical Trials
10,279,722 Total Patients Enrolled
Boston Children's HospitalOTHER
758 Previous Clinical Trials
5,579,294 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,820 Previous Clinical Trials
47,299,421 Total Patients Enrolled

Media Library

G1XCGD Gene Therapy (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02234934 — Phase 1 & 2
Chronic Granulomatous Disease Research Study Groups: Lentiviral G1XCGD Gene Therapy, Part A, Lentiviral G1XCGD Gene Therapy, Part B
Chronic Granulomatous Disease Clinical Trial 2023: G1XCGD Gene Therapy Highlights & Side Effects. Trial Name: NCT02234934 — Phase 1 & 2
G1XCGD Gene Therapy (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02234934 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this study still looking for more participants?

"Unfortunately, this clinical trial is not presently enrolling patients. Although the last update on the website was on 8/1/2022, this could change in the future. There are 23 other trials that may have openings for participants."

Answered by AI
~4 spots leftby Dec 2026