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Clinical trialNCT02161380CompletedPhase 1Interventional

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

SponsorByron LamEnrollment28Leber's Hereditary Optic Neuropathy
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.
clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

  • Byron Lam, MD

    Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami, Miami, FL 33136

    Principal Investigator
    clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • Bascom Palmer Eye Institute, University of Miami

    Miami, Florida, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Injection of scAAV2-P1ND4v2 1.0 X10e11vgInjection of scAAV2-P1ND4v2 1.18x10e9 vgInjection of scAAV2-P1ND4v2 2.4 X10e10vgInjection of scAAV2-P1ND4v2 5.81 X10e9 vg
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
14 Jul 2014
Primary completion
31 Mar 2023
Completion
31 Mar 2025
Last update posted
5 Jun 2025
Registered enrollment
28
Registry id
NCT02161380
clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.