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

NCT02161380 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2025-06-05

Study results available
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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.

Conditions

  • Leber's Hereditary Optic Neuropathy

Interventions

DRUG

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

injection of Total Volume of each intravitreal injection is 200 µL

DRUG

injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med)

injection of Total Volume of each intravitreal injection is 200 µL

DRUG

injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)

injection of Total Volume of each intravitreal injection is 100 µL

DRUG

injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)

injection of Total Volume of each intravitreal injection is 100 µL

Sponsors & Collaborators

  • National Eye Institute (NEI)

    collaborator NIH

  • Byron Lam

    lead OTHER

Principal Investigators

  • Byron Lam, MD · Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami, Miami, FL 33136

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-07-14
Primary Completion
2023-03-31
Completion
2025-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02161380 on ClinicalTrials.gov