Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
Public ClinicalTrials.gov record NCT02161380. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Study identification
- NCT ID
- NCT02161380
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Byron Lam
- Other
- Enrollment
- 28 participants
Conditions and interventions
Conditions
Interventions
- injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher) Drug
- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low), Drug
- injection of scAAV2-P1ND4v2 2.4 X10e10vg (High) Drug
- injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med) Drug
Drug
Eligibility (public fields only)
- Age range
- 15 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 13, 2014
- Primary completion
- Mar 30, 2023
- Completion
- Mar 30, 2025
- Last update posted
- Jun 4, 2025
2014 – 2025
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Bascom Palmer Eye Institute, University of Miami | Miami | Florida | 33136 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02161380, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 4, 2025 · Synced May 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02161380 live on ClinicalTrials.gov.