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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy


NCTID NCT02161380 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Leber's Hereditary Optic Neuropathy
Disease Ontology Term DOID:705
Compound Name ScAAV2-P1ND4v2
Sponsor Byron Lam
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 28
Results Posted View Results

Therapy Information


Target Gene/Variant ND4
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type
Dose 1 5.0E8 vg
Dose 2 2.5E9 vg
Dose 3 1.0E10 vg
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2014-06-06
Completion Date 2025-03-31
Last Update 2024-09-04

Participation Criteria


Eligible Age >=15 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
Recent Updates Safe but low efficacy, researchers did not advance development

Resources/Links