MedTrace Logo

Gene Therapy for X-linked Severe Combined Immunodeficiency

NCT01410019 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-03-11

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

OTHER

Gene transfer

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

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Alain Fischer, MD, PhD · Assistance Publique - Hôpitaux de Paris

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
12 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-12-31
Primary Completion
2015-06-16
Completion
2015-06-16

Countries

  • France

Study Locations

More Related Trials

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 NCT01410019 on ClinicalTrials.gov