CL20-i4-EF1α-hγc-OPT for SCID (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

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 10 years post gene transfer

Awards & highlights

No Placebo-Only Group

Summary

This study is evaluating whether a new gene therapy may help treat a genetic disorder of the immune system.

Eligible Conditions

SCID

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 10 years post gene transfer

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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 study objectives

B-cell function evaluated by Immune response

Event-free survival (EFS)

Number of NK cells

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 4

~1710

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