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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 Batten Disease (Late Infantile Neuronal Ceroid Lipofuscinosis)
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
Dose 1 5 x 10^14 vg
Dose 2 1 x 10^15 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 2023-11-28

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