Status:
UNKNOWN
Gene Therapy for X Linked Severe Combined Immunodeficiency
Lead Sponsor:
Children's Hospital of Chongqing Medical University
Conditions:
Gene Therapy
Eligibility:
MALE
Up to 18 years
Phase:
NA
Brief 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 deficienc...
Eligibility Criteria
Inclusion
- X-SCID patients diagnosed by IL2RG single gene mutation
- No HLA（human 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
- The patient has diagnosed with hematological malignant diseases
- Received chemotherapy within 3 months
- HIV infection or HBV（hepatitis 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
Key Trial Info
Start Date :
May 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2025
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04286815
Start Date
May 1 2020
End Date
May 1 2025
Last Update
March 27 2020
Active Locations (1)
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1
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China, 400014