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Gene Therapy

CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT for Combined Immunodeficiency Diseases (LVXSCID-OC Trial)

Phase 1 & 2
Waitlist Available
Led By Suk See DeRavin, M.D., Ph.D
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up measured through year 15
Awards & highlights

LVXSCID-OC Trial Summary

This trial is testing a new treatment for X-linked severe combined immunodeficiency (XSCID), a disorder that significantly impairs the immune system. The goal is to see if the treatment is safe and effective at restoring immune function.

Eligible Conditions
  • Combined Immunodeficiency Diseases

LVXSCID-OC Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~measured through year 15
This trial's timeline: 3 weeks for screening, Varies for treatment, and measured through year 15 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Distribution of integration sites of the lentiviral vector in reconstituted peripheral blood cells
Incidence of serious side effects due to lentiviral gene transfer
Level of autologous transduced T-lymphocytes with functional γc

LVXSCID-OC Trial Design

1Treatment groups
Experimental Treatment
Group I: Gene-Modified CD34+ HSCsExperimental Treatment3 Interventions
Participants will receive palifermin on Days -6, -5, and -4 and then busulfan on Days -3 and -2. On Day 0, participants will undergo the gene transfer treatment with infusion of the gene-modified CD34+ HSCs. They will receive palifermin on Days 1, 2, and 3.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Palifermin
FDA approved
Busulfan
FDA approved

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,270 Previous Clinical Trials
5,485,224 Total Patients Enrolled
Suk See DeRavin, M.D., Ph.DPrincipal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
2012. "Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03315078.
~1 spots leftby Apr 2025
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