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Gene Therapy Trial Report

Summary

SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection


NCTID NCT03217617 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name Ivlv-X1 lentiviral vector
Sponsor Shenzhen Geno-Immune Medical Institute
Funder Type Other
Recruitment Status
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant IL2RG
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type LV
Dose 1 1E9 vg/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-07-03
Completion Date 2027-12-31
Last Update 2025-09-09

Participation Criteria


Eligible Age 1 Month - 1 Year
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: 1. Diagnosis of SCID-X1 based on: * A proven mutation in the common interleukin-2 receptor gamma chain gene as defined by direct sequencing of patient DNA. * T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigen stimulation. 2. No available HLA identical related donor. 3. With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection. 4. No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia. 5. No prior allogeneic stem cell transplantation. 6. Life expectancy ≥ 3 months. 7. Documented to be negative for HIV infection. 8. Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: 1. No available molecular diagnosis confirming SCID-X1. 2. Existence of an available HLA-identical related donor. 3. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease. 4. Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month). 5. Patients with evidence of infection with HIV-1 or 2. 6. Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical treatment. 7. Current treatment with any immunosuppressive agent, excluding corticosteroids. 8. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.