← Back to Search

Gene Therapy

Gene Therapy for Leber's Optic Atrophy

Q: What are the objectives of this investigation?

<p>This medical trial will last for a duration of 52 weeks and its primary objective is to assess the frequency of adverse events. Secondary goals include measuring improvement in visual acuity, retinal nerve fiber thickness, and Quality-of-Life as assessed by SF-36 questionnaire scores. The ETDRS Visual Acuity Charts will be used to determine if there was an improvement ≥ 0.2 LogMAR or ≥ 0.1 LogMAR from baseline in BCVA; OCT technology will ascertain changes in RNFL; while SF-36's 36 questions across 8 domains aim to gauge patients' general health perceptions on physical activities, social activities</p>

Phase 1 & 2

Recruiting

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

Study 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.Check my eligibility

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.See study design

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

Select...

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

Select...

I am between 18 and 75 years old.

Select...

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

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 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

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

Find a Location

Who is running the clinical trial?

Neurophth Therapeutics IncLead Sponsor

1 Previous Clinical Trials

12 Total Patients Enrolled

Frequently Asked Questions

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

Are there any vacancies available for patients to join this medical experiment?

"Clinicaltrials.gov confirms that this clinical trial - which was inaugurated on August 15th 2023 - is currently in search of participants. The most recent edition to the study materialized on December 3rd 2023."

Answered by AI

Are persons aged over 20 eligible to participate in this clinical trial?

"The stated age range for qualification in this trial is 18-75 years old."

Answered by AI

How many participants are allowed to join in this experiment?

"Affirmative. The information posted on clinicaltrials.gov states that this research study is in the process of enrolling 18 individuals from 3 different medical centres. This trial was first advertised on August 15th 2023 and has been amended most recently on December 3rd 2023."

Answered by AI

Can I qualify to partake in this investigation?

"This medical trial is seeking 18 patients between the ages of adulthood and 75 with Leber Hereditary Optic Neuropathy (LHON). To be considered, they must meet a set of criteria: age ≥18 & ≤75 years old; presence of G3460A mutation in ND1 gene; no other primary LHON associated mutations in mtDNA (ND4 [G11778A] or ND6 [T14484C]); vision loss for > 6 months but < 10 yrs prior to screening; pupils dilated adequately for examination/testing; Hand Motion visual acuity (VA) ≤ 2.3 LogMAR"

Answered by AI

What are the objectives of this investigation?

"This medical trial will last for a duration of 52 weeks and its primary objective is to assess the frequency of adverse events. Secondary goals include measuring improvement in visual acuity, retinal nerve fiber thickness, and Quality-of-Life as assessed by SF-36 questionnaire scores. The ETDRS Visual Acuity Charts will be used to determine if there was an improvement ≥ 0.2 LogMAR or ≥ 0.1 LogMAR from baseline in BCVA; OCT technology will ascertain changes in RNFL; while SF-36's 36 questions across 8 domains aim to gauge patients' general health perceptions on physical activities, social activities"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

2023. "Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05820152.

All > Anterior Ischemic Optic Neuropathy > Neuropathy