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NCT05293626 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations
A Phase 1 study of Leber Hereditary Optic Neuropathy (LHON), sponsored by Neurophth Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 3
- Study locations
NCT05293626: Active Phase 1 study of Leber Hereditary Optic Neuropathy (LHON), sponsored by Neurophth Therapeutics.
NCT05293626 is a Phase 1 study of Leber Hereditary Optic Neuropathy (LHON) that is active but no longer recruiting, run by Neurophth Therapeutics. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05293626, a Phase 1 study of Leber Hereditary Optic Neuropathy (LHON), is active but no longer recruiting, sponsored by Neurophth Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 3
- Study locations
Study Summary
The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation. This study will enroll subjects aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NR082 to evaluate its safety and efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND4 mutation, with laboratory test showing G11778A mutation (a CLIA-certified laboratory) and reduced visual acuity lasted for \> 6 months and \< 10 years.
Primary Outcome
Incidence of adverse events (AEs) within 52 weeks of NR082 intravitreal injection at different doses
Conditions Studied
Interventions
- DRUG NR082 Injection
- DEVICE Injection needle
Study Locations (3)
California
- Stanford Byers Eye Institute - Palo Alto
Colorado
- University of Colorado Health Eye Center - Aurora
Pennsylvania
- Wills Eye Hospital, Neuro Ophthalmology Department - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2023-05-22 |
| Est. Completion | 2029-12 |
| Phase | Phase 1 |
What the registry record for NCT05293626 still lists
NCT05293626 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 1 condition, with Leber Hereditary Optic Neuropathy (LHON) appearing as the primary indexed condition, and to 2 interventions - of which NR082 Injection is the first listed.
NCT05293626 reports a single indexed study location in California, Colorado, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT05293626 about?
NCT05293626 is a clinical study titled "Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations". The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation. This study will enroll subjects aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NR082 to eval...
What is the current status of trial NCT05293626?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2023-05-22. Estimated completion is 2029-12.
What conditions does trial NCT05293626 study?
This clinical trial studies the following conditions: Leber Hereditary Optic Neuropathy (LHON).
What interventions are being tested in trial NCT05293626?
The interventions under investigation include: NR082 Injection (DRUG), Injection needle (DEVICE).
Who is sponsoring clinical trial NCT05293626?
This trial is sponsored by Neurophth Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05293626 being conducted?
This trial has 3 study locations across California, Colorado, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05293626's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 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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