Gene Therapy for Leber's Optic Atrophy
(LHON Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety of a new gene therapy, scAAV2-P1ND4v2, for individuals with Leber's Hereditary Optic Neuropathy (LHON), a condition that causes vision loss. The therapy uses a special viral vector to deliver a gene that helps mitochondria in eye cells function properly. Researchers are testing different doses to determine the safest and most effective one. Individuals with LHON who have the G11778A genetic mutation and experience severe vision loss in one or both eyes may be suitable candidates for this study. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
In a previous study, researchers found that the gene therapy treatment scAAV2-P1ND4v2 was generally well-tolerated. Most participants experienced only mild side effects, easily managed with standard eye treatments. Other research has shown that the treatment helps protect vision without causing major problems. While scAAV2-P1ND4v2 remains under study, early findings are promising for its safety in humans.12345
Why do researchers think this study treatment might be promising for Leber's Optic Atrophy?
Researchers are excited about scAAV2-P1ND4v2 for Leber's Optic Atrophy because it uses gene therapy to address the genetic root of the condition. Unlike current treatments that focus mainly on managing symptoms, this therapy introduces a modified virus to deliver a healthy copy of the ND4 gene directly into retinal cells, potentially halting or even reversing vision loss. This targeted approach could offer more lasting improvements in vision compared to existing options, which don't address the underlying genetic cause. Additionally, the therapy is being tested across various doses to find the most effective and safe option for different stages of vision loss.
What evidence suggests that this gene therapy might be an effective treatment for Leber's Optic Atrophy?
Research has shown that scAAV2-P1ND4v2, a type of gene therapy, is being tested to treat Leber's Hereditary Optic Neuropathy (LHON), a condition that causes vision loss. The trial includes various treatment arms with different dosages of scAAV2-P1ND4v2 to evaluate its effectiveness and safety. This therapy replaces a faulty gene linked to this vision problem. Studies in animals, such as rodents and primates, have demonstrated the treatment's safety. Early results also suggest it might help prevent further vision loss. While these findings are encouraging, more research is needed to confirm its effectiveness in people.36789
Who Is on the Research Team?
Byron Lam, MD
Principal Investigator
Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami, Miami, FL 33136
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravitreal injection of scAAV2-P1ND4v2 at varying doses to assess safety and tolerability
Follow-up
Participants are monitored for safety and effectiveness after treatment, including ocular and systemic toxicity assessments
What Are the Treatments Tested in This Trial?
Interventions
- scAAV2-P1ND4v2
Trial Overview
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.
How Is the Trial Designed?
4
Treatment groups
Experimental Treatment
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.
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
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.
Participants with Chronic Bilateral Severe Vision Loss were administered 100 µL of scAAV2-P1ND4v2 containing a dose of 2.40x10e10 vg.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Byron Lam
Lead Sponsor
National Eye Institute (NEI)
Collaborator
Citations
NCT02161380 | Safety Study of an Adeno-associated Virus ...
The purpose of this dose-escalation study is to assess the safety and tolerability of scAAV2-P1ND4v2 (abbreviated as AAV-ND4) gene replacement therapy.
LHON Gene Therapy Vector Prevents Visual Loss and Optic ...
Discussion. Our results here demonstrated that intravitreal injection of ScAAV2-P1ND4v2 was well tolerated in normal rodents and nonhuman primates and in a ...
Meta-analysis of treatment outcomes for patients with m. ...
Our aim was to assess the visual outcomes of patients with Leber hereditary optic neuropathy (LHON) harboring the m.11778G>A MT-ND4 mutation who had no ...
4.
clinicaltrial.be
clinicaltrial.be/en/details/11797?per_page=20&only_recruiting=0&only_eligible=0&only_active=0Safety Study of an Adeno-associated Virus Vector for Gene...
The purpose of this dose-escalation study is to assess the safety and tolerability of scAAV2-P1ND4v2 (abbreviated as AAV-ND4) gene ...
Safety and Effects of the Vector for the Leber Hereditary ...
Koilkonda et al demonstrate the safety and effects of the gene therapy vector to be used in a proposed gene therapy clinical trial for Leber ...
NCT02161380 | Safety Study of an Adeno-associated Virus ...
The purpose of this dose-escalation study is to assess the safety and tolerability of scAAV2-P1ND4v2 (abbreviated as AAV-ND4) gene replacement therapy in ...
Safety and Effects of the Vector for the Leber Hereditary ...
These findings indicate that WT allotopic ND4 gene therapy prevents visual loss and RGC dysfunction with gene delivery using the highly efficient expression ...
Gene Therapy Trial Report
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy ; Compound Name, ScAAV2-P1ND4v2 ; Sponsor, Byron Lam.
Safety of Lenadogene Nolparvovec Gene Therapy Over 5 ...
The product was well tolerated, with mostly mild ocular side effects responsive to conventional ophthalmologic treatments. Keywords. Leber hereditary optic ...
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