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Clinical Trial Report

Gene Therapy Trial Report

Summary

Study for the Treatment for CLN7 Disease


NCTID NCT04737460 (View at clinicaltrials.gov)
Description
Indication CLN7
Compound Name AAV9/CLN7
Sponsor Benjamin Greenberg
Funder Type Other
Status
Active not recruiting
Enrollment Count 4

Therapy Information


Target Gene/Variant MFSD8
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 5E14 vg
Dose 2 1E15 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2021-01-15
Completion Date 2029-02-01
Last Update 2024-12-13

Participation Criteria


Eligible Age 1 Year - 18 Years
Standard Ages Child, Adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
Recent Updates

Resources/Links