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Clinical Trial Report

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
Recruiting
Enrollment Count 10
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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type LV
Editor Type
Dose 1 1E9 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Na
Submit Date 2018-07-17
Completion Date 2027-12-31
Last Update 2024-07-17

Participation Criteria


Eligible Age >=1 Month
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

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.