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Gene Therapy Trial Report

Summary

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)


NCTID NCT03645460 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Disease Ontology Term DOID:5810
Compound Name ADA 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 ADA
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 Na
Submit Date 2018-07-17
Completion Date 2027-12-31
Last Update 🔄 2026-06-23

Participation Criteria


Eligible Age >=1 Month
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Diagnosis of classical ADA-SCID based on: * A proven defective adenosine deaminase (ADA) 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 antigens. * With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection. * No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children. * No prior allogeneic stem cell transplantation. * Life expectancy ≥ 2 months. * Negative for HIV infection. * Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: * None
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.