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Gene Therapy for X Linked Severe Combined Immunodeficiency

NCT04286815 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2020-03-27

No results posted yet for this study

Summary

A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.

Conditions

  • Gene Therapy

Interventions

DEVICE

Lentiviral Vector Gene Therapy

Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells

Sponsors & Collaborators

  • Children's Hospital of Chongqing Medical University

    lead OTHER

Principal Investigators

  • Xiaodong Zhao, PHD · Assistant President of Children's Hospital of Chongqing Medical University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-05-01
Primary Completion
2023-05-01
Completion
2025-05-01

Countries

  • China

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 NCT04286815 on ClinicalTrials.gov