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Gene Therapy

CL20-i4-EF1α-hγc-OPT for Severe Combined Immunodeficiency (LVXSCID-ND Trial)

Phase 1 & 2
Waitlist Available
Led By Stephen Gottschalk, MD
Research Sponsored by St. Jude Children's Research Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Availability of a HLA matched sibling for allogeneic transplantation
Prior therapy with allogeneic stem cell transplantation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 10 years post gene transfer
Awards & highlights

LVXSCID-ND Trial Summary

This trial is testing a new way to treat SCID-X1, a genetic disorder that prevents the development of a functioning immune system. The new method, called lentiviral gene transfer, involves transferring a normal copy of the common gamma chain gene into the patient's bone marrow stem cells. The investigators want to determine if the procedure is safe and effective.

Eligible Conditions
  • X-Linked Severe Combined Immunodeficiency (SCID)

LVXSCID-ND Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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You do not have a sibling who is a match for a transplant.
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You have received a stem cell transplant from someone else before.

LVXSCID-ND Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 10 years post gene transfer
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 10 years post gene transfer for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of patients with adequate cell collection and processing
Number of patients with adequate neutrophil count recovery after busulfan conditioning
Number of patients with successful reconstitution
+2 more
Other outcome measures
B-cell function evaluated by Immune response
Event-free survival (EFS)
Number of NK cells
+4 more

Side effects data

From 2021 Phase 2 trial • 22 Patients • NCT01203020
41%
Death
100%
80%
60%
40%
20%
0%
Study treatment Arm
Allogeneic Hematopoietic Progenitor Cell Transplant

LVXSCID-ND Trial Design

1Treatment groups
Experimental Treatment
Group I: TreatmentExperimental Treatment3 Interventions
Participants will undergo a bone marrow harvest in the operating room to obtain bone marrow cells. Cells will be isolated and purified utilizing the CliniMacs device. These cells will undergo vector transduction with the lentiviral vector that contains a normal copy of the γc gene gene (CL20-i4-EF1α-hγc-OPT) and then the transduced cells will be reinfused back into the patient. Participants will receive a conditioning regimen of busulfan 3 days prior and 2 days prior to infusion of vector-corrected cells.intervention: CL20-i4-EF1α-hγc-OPT
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)NIH
3,810 Previous Clinical Trials
47,290,618 Total Patients Enrolled
3 Trials studying Severe Combined Immunodeficiency
106 Patients Enrolled for Severe Combined Immunodeficiency
St. Jude Children's Research HospitalLead Sponsor
426 Previous Clinical Trials
5,306,577 Total Patients Enrolled
3 Trials studying Severe Combined Immunodeficiency
1,008 Patients Enrolled for Severe Combined Immunodeficiency
Assisi FoundationOTHER
10 Previous Clinical Trials
363 Total Patients Enrolled

Frequently Asked Questions

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

Are patients still being enrolled in this research project?

"Yes, as of now this study is still recruiting patients. The clinical trial was initially posted on August 17th, 2016 and updated January 5th, 2022. They are looking for a total of 28 patients that will be split between 3 locations."

Answered by AI

What are the results of using CL20-i4-EF1α-hγc-OPT for treating patients?

"CL20-i4-EF1α-hγc-OPT can be an effective treatment for various conditions including allogeneic hematopoietic stem cell transplant, chronic myelogenous leukemia, conditioning regimens for allogeneic stem cell transplantation therapy, and thrombocythemia, essential."

Answered by AI

How many individuals are taking part in this test?

"The clinical trial is accepting patients at this time, as evidenced by information available on clinicaltrials.gov. This research was originally posted on 8/17/2016 and updated most recently on 1/5/2022. The study requires 28 individuals from 3 different locations."

Answered by AI

Are there any similar treatments to CL20-i4-EF1α-hγc-OPT that have been looked into?

"Currently, there are one hundred and four independent clinical trials assessing the efficacy of CL20-i4-EF1α-hγc-OPT. Of these investigations, thirteen have progressed to Phase 3. Most of the related research is emanating out of Philadelphia, Pennsylvania; although, there are 1072 locations running clinical trials for CL20-i4-EF1α-hγc OPT globally."

Answered by AI
~4 spots leftby Aug 2025