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Gene Therapy

Gene Therapy for Leber's Optic Atrophy

Phase 1 & 2

Waitlist Available

Research Sponsored by Neurophth Therapeutics Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

The genotype testing result shows the presence of G11778A mutation in the ND4 gene, and the absence of the other primary LHON associated mutations in the mitochondrial DNA (mtDNA) (confirmed by a CLIA-certified international laboratory

Each eye of the subject must maintain at least Hand Motion VA (≤ 2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for refraction and VA examinations) in this study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at weeks 2, 6,12, 26, 40, 52, 78, 104, 156, 208, and 260

Awards & highlights

Study Summary

This trial tests the safety/effectiveness of a single eye injection for patients with Low-Hereditary Optic Neuropathy caused by the ND4 mutation. Patients must be 18-75, with reduced vision for 6-10 months.

Who is the study for?

Adults aged 18-75 with Leber's Hereditary Optic Neuropathy (LHON) due to ND4 gene mutations, who have experienced vision loss for more than 6 months but less than 10 years. Participants must not be pregnant or breastfeeding and agree to use contraception for at least six months post-treatment. They should not have other eye diseases affecting vision or a history of certain treatments like ocular gene therapy.Check my eligibility

What is being tested?

The trial is testing the safety and effectiveness of NR082, administered through a single intravitreal injection in one eye, for treating LHON caused by ND4 mutations. The goal is to see if this treatment can improve visual acuity in affected individuals.See study design

What are the potential side effects?

Potential side effects may include typical risks associated with intravitreal injections such as eye inflammation, increased intraocular pressure, bleeding inside the eye, cataract formation, retinal detachment, infection risk (endophthalmitis), and possible allergic reactions to the components of NR082.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My genetic test shows I have the G11778A mutation in ND4, without other LHON mutations.

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I can see hand movements with both eyes.

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I have vision loss from LHON with some vision of 0.5 LogMAR or better.

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I am between 18 and 75 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at weeks 2, 6,12, 26, 40, 52, 78, 104, 156, 208, and 260

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at weeks 2, 6,12, 26, 40, 52, 78, 104, 156, 208, and 260

This trial's timeline: 3 weeks for screening, Varies for treatment, and at weeks 2, 6,12, 26, 40, 52, 78, 104, 156, 208, and 260 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Incidence of adverse events (AEs)

Incidence of dose-limiting toxicities (DLT)

Incidence of serious adverse events (SAEs)

Secondary outcome measures

Change from baseline in retinal ganglion cell complex thickness of the study eye

Change from baseline in retinal nerve fiber layer (RNFL) thickness of the study eye

Change from baseline in spatial frequency parameters of contrast sensitivity in the study eye

+14 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: NR082 injectionExperimental Treatment2 Interventions

Potential doses at the dose-finding stage: 0.5E9 vg, 0.05 mL/eye/dose (low dose) 1.5E9 vg, 0.05 mL/eye/dose (starting dose) 3.0E9 vg, 0.05 mL/eye/dose (intermediate dose) 4.5E9 vg, 0.05 mL/eye/dose (high dose)

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Neurophth Therapeutics IncLead Sponsor

1 Previous Clinical Trials

18 Total Patients Enrolled

Frequently Asked Questions

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

Is eligibility for this investigation restricted to individuals aged 18 or older?

"In order to be considered for this trial, the age requirements are 18 years old minimum and 75 years old maximum."

Answered by AI

Is there still availability for potential participants in this trial?

"This clinical trial, which was initially advertised on May 22nd 2023, is still recruiting participants according to the information available on clinicaltrials.gov. The last update took place on October 22nd of the same year."

Answered by AI

What is the total number of participants involved in this clinical trial?

"Affirmative, the clinicaltrials.gov portal reveals that this experimental trial is actively recruiting participants. The research was first posted on May 22nd 2023 with a recent update occuring October 22nd of the same year. At present, 18 test subjects across 3 sites are needed for participation."

Answered by AI

What is the primary purpose of this clinical investigation?

"The primary endpoint for this 52 week clinical trial is to observe the incidence of Serious Adverse Events (SAEs). Other outcomes include evaluating Mean change from baseline in BCVA (LogMAR) via ETDRS visual acuity charts and Change from baseline in Pattern Standard Deviation (PSD) using Humphrey static perimetry. Additionally, alterations in function of different regions of the retina will be evaluated with HVF analyzers."

Answered by AI

Is my participation in this trial permissible?

"This clinical trial seeks to recruit 18 individuals with Leber Hereditary Optic Neuropathy (LHON) that are aged between eighteen and seventy-five. To be eligible, patients must satisfy the following criteria: Be of legal age for consent at screening; demonstrate vision loss due to LHON with a BCVA ≥ 0.5 LogMAR; harbour the G11778A mutation in the ND4 gene whilst lacking other primary LHON associated mutations in mitochondrial DNA; present with visual acuity impairment lasting greater than six months but less than ten years prior to enrolment; maintain Hand Motion VA ≤ 2.3 LogMAR throughout ocular"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

2023. "Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05293626.

All > Anterior Ischemic Optic Neuropathy > Neuropathy