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Virus Therapy

Gene Therapy for Leber's Optic Atrophy (LHON Trial)

Phase 1
Waitlist Available
Led By Byron Lam, MD
Research Sponsored by Byron Lam
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 15 or older;
Ability to perform tests of visual and retinal function
Must not have
History of systemic diseases having ocular manifestations likely to confound assessment of study results
Type I diabetes or the presence of diabetic retinopathy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights

Summary

This trial is testing whether or not a new treatment is safe for people. The hypothesis is that the treatment will not cause any toxicity that would lead to blindness.

Who is the study for?
This trial is for individuals aged 15 or older with Leber's Hereditary Optic Neuropathy (LHON) and a specific genetic mutation (G11778A). Participants must be in good health, able to perform visual tests, comply with study procedures, and provide informed consent. Exclusions include other eye diseases, severe liver issues, blood disorders, previous eye surgery on the affected eye, pregnancy/nursing women not using contraception, certain systemic or autoimmune diseases.
What is being tested?
The trial is testing different doses of an injection called scAAV2-P1ND4v2 to treat LHON. There are four dose levels: low, medium-higher and high. The main goal is to check if these injections are safe and do not cause any loss of vision in the treated eyes.
What are the potential side effects?
While the primary concern is potential loss of vision from toxicity related to the treatment itself as it's a gene therapy study; detailed side effects aren't provided but may include typical reactions at injection sites such as pain or inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 15 years old or older.
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I can undergo tests for my eyesight and retina.
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I have LHON with the G11778A mutation confirmed by a genetic test.
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I am 15 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a disease that affects my eyes and could interfere with the study's results.
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I have Type I diabetes or diabetic eye damage.
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I cannot see any light with either of my eyes.
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I had cancer other than skin cancer more than 5 years ago and it was cured.
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I have no eye conditions except for needing glasses or contacts.
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I have a history of conditions like multiple sclerosis or Parkinson's.
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I have had eye surgery on the eye that will receive the injection.
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I am allergic to eye drops used for dilating pupils or have a condition that makes dilation unsafe.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Treatment Related Adverse Events
Secondary study objectives
Best-corrected Visual Acuity

Trial Design

4Treatment groups
Experimental Treatment
Group I: Medium dose (5.81x10e9 vg)Experimental Treatment1 Intervention
Participants with Chronic Bilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 5.81x10e9 vg. Participants with Acute Bilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 5.81x10e9 vg. Participants with Acute Unilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 5.81x10e9 vg.
Group II: Low-dose (1.18x10e9 vg)Experimental Treatment1 Intervention
Participants with Chronic Bilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 1.18x10e9 vg. Participants with Acute Bilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 1.18x10e9 vg. Participants with Acute Unilateral Severe Vision Loss were administered 200 µL of scAAV2-P1ND4v2 containing a dose of 1.18x10e9 vg
Group III: Higher dose (1x10e11vg)Experimental Treatment1 Intervention
Participants with Chronic Bilateral Severe Vision Loss were administered 100 µL of scAAV2-P1ND4v2 containing a dose of 1x10e11vg. Participants with Acute Bilateral Severe Vision Loss were administered 100 µL of scAAV2-P1ND4v2 containing a dose of 1x10e11vg. Participants with Acute Unilateral Severe Vision Loss were administered 100 µL of scAAV2-P1ND4v2 containing a dose of 1x10e11vg.
Group IV: High dose (2.40x10e10 vg)Experimental Treatment1 Intervention
Participants with Chronic Bilateral Severe Vision Loss were administered 100 µL of scAAV2-P1ND4v2 containing a dose of 2.40x10e10 vg.

Find a Location

Who is running the clinical trial?

Byron LamLead Sponsor
1 Previous Clinical Trials
6 Total Patients Enrolled
National Eye Institute (NEI)NIH
552 Previous Clinical Trials
1,406,960 Total Patients Enrolled
Byron Lam, MDPrincipal InvestigatorBascom Palmer Eye Institute, Miller School of Medicine, University of Miami, Miami, FL 33136

Media Library

scAAV2-P1ND4v2 (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02161380 — Phase 1
Leber Optic Neuropathy Research Study Groups: Low-dose (1.18x10e9 vg), High dose (2.40x10e10 vg), Higher dose (1x10e11vg), Medium dose (5.81x10e9 vg)
Leber Optic Neuropathy Clinical Trial 2023: scAAV2-P1ND4v2 Highlights & Side Effects. Trial Name: NCT02161380 — Phase 1
scAAV2-P1ND4v2 (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02161380 — Phase 1
~3 spots leftby Oct 2025
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