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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
Development Status Active
Indication Giant Axonal Neuropathy
Disease Ontology Term DOID:0090068
Compound Name ScAAV9/JeT-GAN
Compound Alias TSHA-120
Sponsor National Institute of Neurological Disorders and Stroke (NINDS)
Funder Type NIH
Recruitment Status
Active not recruiting
Enrollment Count 14 (ACTUAL)
Results Posted Not Available

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 none
Dose 1 3.5E13 vg (n=2)
Dose 2 1.2E14 vg (n=4)
Dose 3 1.8E14 vg (n=5)
Dose 4 3.5E14 vg (n=3)
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2015-02-12
Completion Date 2035-04-01
Last Update 2025-09-30

Participation Criteria


Eligible Age 3 Years - 99 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Taysha discontinued development in September 2023, transferred development rights to NIH

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