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Gene Therapy Trial Report

Summary

Gene Therapy for X Linked Severe Combined Immunodeficiency


NCTID NCT04286815 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name Lentiviral vector/IL2RG
Sponsor Children's Hospital of Chongqing Medical University
Funder Type Other
Recruitment Status
Unknown
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant IL2RG
Therapy Type Gene transfer
Therapy Route Undisclosed
Mechanism of Action Functional gene replacement
Route of Administration Undisclosed
Drug Product Type Undisclosed
Delivery System Undisclosed
Vector Type undisclosed
Dose 1 Undisclosed

Study Record Dates


Current Stage Na
Submit Date 2020-02-23
Completion Date 2025-05-01
Last Update 2020-03-27

Participation Criteria


Eligible Age <=18 Years
Standard Ages Child, Adult
Sexes Eligible for Study MALE
Eligibility Criteria
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.