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Gene Therapy

Gene Therapy for Leber's Hereditary Optic Neuropathy (REFLECT Trial)

Phase 3
Waitlist Available
Led By Nancy Newman, MD
Research Sponsored by GenSight Biologics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.
Age 15 years or older on the date of signed informed consent.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1.5-year and 2-years post baseline treatment
Awards & highlights

REFLECT Trial Summary

This trial is testing a gene therapy called GS010 to see if it can improve the retina function and structure in people who have a certain kind of mitochondrial disease.

Who is the study for?
This trial is for people aged 15 and older with vision loss from Leber's Hereditary Optic Neuropathy (LHON) due to the ND4 mutation, within a year of losing vision. Participants must not have other optic nerve mutations, severe visual impairment beyond hand motion acuity, or active eye inflammation. They can't be using idebenone or have a history of substance abuse.Check my eligibility
What is being tested?
The study tests GS010 gene therapy against a placebo to see if it improves vision in LHON patients by injecting it into both eyes. It aims to determine how safe and effective this treatment is over up to one year.See study design
What are the potential side effects?
Potential side effects may include typical risks associated with intravitreal injections such as eye inflammation, infection risk increase, possible allergic reactions to components of the therapy, and discomfort at the injection site.

REFLECT Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have had repeated eye inflammation or currently have it.
Select...
I am 15 years old or older.
Select...
I have vision loss in at least one eye due to ND4 LHON.
Select...
I refuse to stop taking idebenone.
Select...
My genetic test shows I have the G11778A mutation in the ND4 gene without other LHON mutations.
Select...
I am 15 years old or older.
Select...
I have vision loss in at least one eye due to ND4 LHON.
Select...
I cannot have eye injections due to medical reasons.

REFLECT Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1.5-year and 2-years post baseline treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1.5-year and 2-years post baseline treatment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Best-Corrected Visual Acuity (BCVA) reported using Log of the Minimal Angle of Resolution (LogMAR) - 1 year
Secondary outcome measures
Best-Corrected Visual Acuity (BCVA) reported with LogMAR - 2 years
Humphrey Visual Field (HVF) parameter
Pelli Robson Low Vision Contrast Sensitivity parameter
+4 more
Other outcome measures
Adverse events (AEs) and serious adverse events (SAEs)
Blood Bio-dissemination of AAV2 Vector DNA
Electrocardiograms
+3 more

Side effects data

From 2022 Phase 3 trial • 62 Patients • NCT03406104
10%
Cataract
8%
Gamma-Glutamyltransferase Increased
6%
Intraocular Pressure Increased
6%
Covid-19
3%
hip fracture
2%
humerus fracture
2%
neoplasm progression
2%
cerebral haemorrage
2%
glioblastoma multiforme
2%
lower limb fracture
2%
pneumonia
2%
appenditis perforated
2%
limb injury
2%
cardiac arrest
2%
ileus
100%
80%
60%
40%
20%
0%
Study treatment Arm
Subjects

REFLECT Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Treatment Arm 1Experimental Treatment1 Intervention
Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 1 will receive intravitreal GS010 in both eyes.
Group II: Treatment Arm 2Placebo Group2 Interventions
Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 2 will receive GS010 in one eye and placebo intravitreal injection in the other eye.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Lenadogene nolparvovec
Not yet FDA approved

Find a Location

Who is running the clinical trial?

GenSight BiologicsLead Sponsor
8 Previous Clinical Trials
228 Total Patients Enrolled
Nancy Newman, MDPrincipal InvestigatorEmory University Hospital Atlanta, Georgia, United States, 30322
1 Previous Clinical Trials
62 Total Patients Enrolled

Media Library

Leber Optic Neuropathy Clinical Trial 2023: GS010 Highlights & Side Effects. Trial Name: NCT03293524 — Phase 3

Frequently Asked Questions

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

If GS010 proves to be unsuccessful, what are the consequences for patients?

"GS010 is a Phase 3 trial medication, meaning that while there is some data supporting efficacy, multiple rounds of data have been collected to support safety."

Answered by AI

Are there any vacancies in this trial for new test subjects?

"According to the latest information available on clinicaltrials.gov, this particular study is not currently looking for any more participants. Although 18 other trials are ongoing, this one was posted on March 12th, 2018 and last edited on August 2nd, 2022."

Answered by AI

In how many different medical clinics is this medical study being conducted today?

"Presently, this clinical trial has 7 enrolling patients from locations such as Emory Healthcare - The Emory Clinic in Atlanta and Vanderbilt Eye Institute in Nashville. There are also other enrolment centres located in Pasadena and 6 other cities."

Answered by AI

Who else is applying?

What state do they live in?
California
New York
How old are they?
18 - 65
What site did they apply to?
Emory Healthcare - The Emory Clinic
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
Recent research and studies
clinicaltrials.govStudy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
2018. "Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03293524.
~2 spots leftby Jun 2024
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