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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 2025-09-09

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 2
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.