Gene Therapy for X Linked Severe Combined Immunodeficiency

  • STATUS
    Recruiting
  • End date
    May 1, 2025
  • participants needed
    10
  • sponsor
    Children's Hospital of Chongqing Medical University
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Updated on 19 February 2024
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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-SCIDsevere 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.

Details
Condition gene therapy
Age 18 years and younger
Treatment Lentiviral Vector Gene Therapy
Clinical Study IdentifierNCT04286815
SponsorChildren's Hospital of Chongqing Medical University
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

X-SCID patients diagnosed by IL2RG single gene mutation
No HLAhuman leukocyte antigen matching donor
Hematopoietic stem cell transplantation failed and the time from transplantation was more than 18 months
Severe and persistent refractory infections
Life expectancy of > : 4 months
HIV PCR in peripheral blood was negative
the children and their families signed informed consent and were willing to enter the clinical trial and complete follow-up

Exclusion Criteria

The patient has diagnosed with hematological malignant diseases
Received chemotherapy within 3 months
HIV infection or HBVhepatitis B virus infection
The patient or his first-degree relative has developed a malignant tumor within the age of 18 or has been diagnosed with malignant tumor prone genes
Although the patient with X-SCID was diagnosed as IL2RG single gene mutation , the clinical phenotype was not severe, so they could continue to wait for the donor search
Patients whose family members have no intention to continue the follow-up treatment in any link
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