NCT02161380

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Byron Lam · Academic / Other
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The purpose of this dose-escalation study is to assess the safety and tolerability of scAAV2-P1ND4v2 (abbreviated as AAV-ND4) gene replacement therapy in subjects confirmed with the G11778A mutation in mtDNA responsible for Leber's Hereditary Optic Neuropathy. Ocular and systemic toxicity will be assessed following vector administration to determine if there are adverse changes that may be associated with vector administration. This first-in-man (FIM) clinical trial will assess the safety, tolerability, and potential efficacy of a single intravitreal injection in patient groups reflecting the acute, pre-symptomatic, and chronic stages and manifestation of the LHON disease.

Conditions

• Leber's Hereditary Optic Neuropathy

Interventions

Type	Name	Description
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

Timeline

• Start date: 2014-07-14
• Primary completion: 2023-03-31
• Completion: 2025-03-31
• First posted: 2014-06-11
• Last updated: 2025-06-05
• Results posted: 2024-08-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study