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

> **NCT02161380** · PHASE1 · COMPLETED · sponsor: **Byron Lam** · enrollment: 28 (actual)

## Conditions studied

- Leber's Hereditary Optic Neuropathy

## Interventions

- **DRUG:** injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),
- **DRUG:** injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med)
- **DRUG:** injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
- **DRUG:** injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)

## Key facts

- **NCT ID:** NCT02161380
- **Lead sponsor:** Byron Lam
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2014-07-14
- **Primary completion:** 2023-03-31
- **Final completion:** 2025-03-31
- **Target enrollment:** 28 (ACTUAL)
- **Last updated:** 2025-06-05

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02161380

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02161380, "Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy". Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/clinical/NCT02161380. Licensed CC0.

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