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Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

NCT01175239 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2017-08-01

No results posted yet for this study

Summary

X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys. This trial aims to treat SCID-X1 patients using gene therapy to replace the defective gene.

Conditions

  • X-linked Severe Combined Immunodeficiency

Interventions

GENETIC

Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre

Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre

Sponsors & Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-04-30
Primary Completion
2018-12-31
Completion
2018-12-31

Countries

  • United Kingdom

Study Locations

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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 NCT01175239 on ClinicalTrials.gov