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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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 years

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.

0 / 12Eligibility criteria met