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Clinical Trial Report

Gene Therapy Trial Report

Summary

Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy


NCTID NCT02362438 (View at clinicaltrials.gov)
Description
Indication Giant Axonal Neuropathy
Compound Name ScAAV9/JeT-GAN
Sponsor Taysha Gene Therapies, Inc.
Funder Type Industry
Status
Active not recruiting
Enrollment Count 21

Therapy Information


Target Gene/Variant GAN
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 3.5 x 10^13 vg
Dose 2 1.2 x 10^14 vg
Dose 3 1.8 x 10^14 vg
Dose 4 3.5 x 10^14 vg
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2015-02-12
Completion Date 2035-04-01
Last Update 2023-11-21

Participation Criteria


Eligible Age 3 Years - 99 Years
Standard Ages Child, Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
Recent Updates

Resources/Links