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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 year

Awards & highlights

LHON Trial 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.Check my eligibility

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.See study design

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.

LHON Trial 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.

LHON Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assessment of Primary Endpoint - Toxicity

Secondary outcome measures

Assessment of Secondary Endpoint - Safety & Efficacy

LHON Trial Design

3Treatment groups

Experimental Treatment

Group I: 3 Acute Unilateral Severe Vision LossExperimental Treatment3 Interventions

injection of scAAV2-P1ND4v2

Group II: 2 Acute Bilateral Severe Vision LossExperimental Treatment3 Interventions

injection of scAAV2-P1ND4v2

Group III: 1 Chronic Bilateral Severe Vision LossExperimental Treatment4 Interventions

injection of scAAV2-P1ND4v2

0 / 4Eligibility criteria met

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

546 Previous Clinical Trials

1,401,900 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: 2 Acute Bilateral Severe Vision Loss, 3 Acute Unilateral Severe Vision Loss, 1 Chronic Bilateral Severe Vision Loss

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the FDA recognize the injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low) as permissible?

"As this is a Phase 1 trial and limited data exists on safety or efficacy, the safety of injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low) was scored at a value of one."

Answered by AI

Is recruitment for this clinical trial ongoing?

"According to the clinicaltrials.gov, this particular medical trial is not in need of participants currently. The inaugural posting was on July 14th 2014 and it has been amended as recently as August 20th 2022. While no longer recruiting patients, there are 18 other trials searching for volunteers right now."

Answered by AI