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NCT02161380 · ClinicalTrials.gov registry record · Phase 1
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
A Phase 1 study, sponsored by Byron Lam.
- Completed
- Registry status
- Phase 1
- Development phase
- 28
- Enrollment target
NCT02161380: Completed Phase 1 study, sponsored by Byron Lam.
NCT02161380 is a Phase 1 study that has completed, run by Byron Lam. The registered enrollment target is 28 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02161380, a Phase 1 study, has completed, sponsored by Byron Lam.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 28 participants
- Enrollment target
Study 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.
Primary Outcome
Number of treatment-related adverse events will be assessed as per investigator with respective to relationship to the investigative product.
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)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 28 participants |
| Start Date | 2014-07-14 |
| Est. Completion | 2025-03-31 |
| Phase | Phase 1 |
What the finished NCT02161380 record still lists
NCT02161380 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 28 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (53% lower).
The record links to 0 conditions, and to 4 interventions - of which injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low), is the first listed.
NCT02161380 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02161380 about?
NCT02161380 is a clinical study titled "Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy". 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.
What is the current status of trial NCT02161380?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 28 participants. The study started on 2014-07-14. Estimated completion is 2025-03-31.
What interventions are being tested in trial NCT02161380?
The interventions under investigation include: 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) (DRUG).
Who is sponsoring clinical trial NCT02161380?
This trial is sponsored by Byron Lam, which has 2 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02161380's enrollment target sits among peer trials
28 1447th of 2000 higher than 538 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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