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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 clinical manifested vision loss due to LHON, and any eye BCVA ≥ 0.5 LogMAR

Age at the time of signing the informed consent form: the age of the subjects must be ≥ 18 years old and ≤ 75 years old Type of Subject and Disease Characteristics

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at weeks 2, 6,12, 26, 40, 52, 78, 104, 130, 156, 182, 208, 234, and 260

Awards & highlights

No Placebo-Only Group

Summary

This trial will test a new treatment for Leber's Hereditary Optic Neuropathy (LHON) caused by a genetic mutation. Participants aged 18-75 will get a single injection in the eye to evaluate the safety, tolerability, and efficacy.

Who is the study for?

This trial is for adults aged 18 to 75 with vision loss from Leber's Hereditary Optic Neuropathy (LHON) due to ND1 gene mutation, present for more than 6 months but less than 10 years. Participants must have a specific genetic mutation (G3460A), and their worst eye's visual acuity should be at least Hand Motion VA. Men must use contraception post-treatment.

What is being tested?

The NFS-02 Injection is being tested in this study. It involves a one-time injection into the eye of individuals with LHON to check its safety, how well it's tolerated, and initial effectiveness in improving vision or slowing down vision loss.

What are the potential side effects?

While not explicitly listed here, potential side effects may include discomfort or complications related to intravitreal injections such as eye inflammation, infection risk increase around the injection site, bleeding inside the eye, retinal detachment or cataracts.

Eligibility Criteria

Inclusion Criteria

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

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

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I am between 18 and 75 years old.

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My genetic test shows I have the G3460A mutation in the ND1 gene without other LHON mutations.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at weeks 2, 6,12, 26, 40, 52, 78, 104, 130, 156, 182, 208, 234, and 260

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at weeks 2, 6,12, 26, 40, 52, 78, 104, 130, 156, 182, 208, 234, and 260

This trial's timeline: 3 weeks for screening, Varies for treatment, and at weeks 2, 6,12, 26, 40, 52, 78, 104, 130, 156, 182, 208, 234, and 260 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of adverse events (AEs)

Incidence of dose-limiting toxicities (DLT)

Incidence of serious adverse events (SAEs)

Secondary study objectives

Change from baseline in contrast sensitivity in the injected eye and non-injected eye

Change from baseline in retinal ganglion cell complex thickness in the injected eye and non-injected eye

Change from baseline in retinal nerve fiber layer (RNFL) thickness in the injected eye and non-injected eye

+12 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: NFS-02 InjectionExperimental Treatment1 Intervention

Potential doses at the dose-finding stage: 5.0×107 vg, 0.05 mL/eye/dose (low dose) 1.5×108 vg, 0.05 mL/eye/dose (starting dose) 5.0×108 vg, 0.05 mL/eye/dose (intermediate dose) 1.5×109 vg, 0.05 mL/eye/dose (high dose)

0 / 3Eligibility criteria met