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Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

NCT01512888 · Status: SUSPENDED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2025-11-10

No results posted yet for this study

Summary

SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system. The purpose of this study is to determine if a new method, called lentiviral gene transfer, can be used to treat SCID-X1. This method involves transferring a normal copy of the common gamma chain gene into the participant's bone marrow stem cells. The investigators want to determine if the procedure is safe, whether it can be done according to the methods they have developed, and whether the procedure will provide a normal immune system for the patient. It is hoped that this type of gene transfer may offer a new way to treat children with SCID-X1 that do not have a brother or sister who can be used as a donor for stem cell transplantation.

Conditions

  • Severe Combined Immunodeficiency Disease, X-linked

Interventions

GENETIC

CL20-i4-EF1α-hγc-OPT

Participants will undergo infusion with autologous CD34+ bone marrow cells transduced with a lentiviral vector that contains a normal copy of the human γc gene.

DRUG

Busulfan

Given intravenously (IV).

DEVICE

CliniMacs

Isolation and purification of CD34+ stem cells will be done after the unmodified frozen backup is obtained and in accordance with our FDA IND and in accordance with the CliniMacs manual of operations.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Assisi Foundation

    collaborator OTHER

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • St. Jude Children's Research Hospital

    lead OTHER

Principal Investigators

  • Stephen Gottschalk, MD · St. Jude Children's Research Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
24 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-08-17
Primary Completion
2026-08-31
Completion
2034-08-31
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01512888 on ClinicalTrials.gov