CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 28 enrolled
Drug / intervention
injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low), +3 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT02161380
NCT02161380Phase 1Completed
CompareCT.gov

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA

Byron Lam·interventional·Posted Jun 11, 2014·Updated Jun 5, 2025

In Brief

A Phase 1 clinical trial evaluating injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), and 2 other interventions for Leber's Hereditary Optic Neuropathy. Completed, enrolled 28 participants across 1 site.

Detailed Summary

The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON patients.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLeber's Hereditary Optic Neuropathy
CountriesUnited States
CollaboratorsNational Eye Institute (NEI)

Timeline

Phase 1CompletedFinished
201520162017201820192020202120222023202420252026
First PostedJun 11, 2014
Enrollment StartJul 14, 2014
Primary CompletionMar 31, 2023
Study CompletionMar 31, 2025
TodayJul 2, 2026
Enrollment to primary: 8.7 yearsPosted 12.1 years ago

Interventions

injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),drug

injection of Total Volume of each intravitreal injection is 200 µL

injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med)drug

injection of Total Volume of each intravitreal injection is 200 µL

injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)drug

injection of Total Volume of each intravitreal injection is 100 µL

injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)drug

injection of Total Volume of each intravitreal injection is 100 µL