Gene Therapy for X Linked Severe Combined Immunodeficiency

Chongqing Medical University

Status: Enrolling

Conditions: Gene Therapy

Treatments: Device: Lentiviral Vector Gene Therapy

Study type: Interventional

Funder types: Other

Identifiers

NCT04286815

CHCMU gene therapy

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: Male

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

1. The patient has diagnosed with hematological malignant diseases
2. Received chemotherapy within 3 months
3. HIV infection or HBV（hepatitis B virus） infection
4. 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
5. 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;
6. Patients whose family members have no intention to continue the follow-up treatment in any link

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

10 participants in 1 patient group

Experimental Group

Experimental group

Description:

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）.

Treatment:

Device: Lentiviral Vector Gene Therapy