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Gene Therapy

GS010 for Leber Optic Neuropathy (RESTORE 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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up nadir to 5 years post-treatment, assessed at baseline in rescue and reverse studies to year 5 post-treatment
Awards & highlights

RESTORE Trial Summary

This trial is testing a gene therapy for people with LHON, a disease that affects the mitochondria. The goal is to see if the therapy is safe and effective long-term, and if it improves quality of life.

Eligible Conditions
  • Leber Optic Neuropathy

RESTORE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~nadir to 5 years post-treatment, assessed at baseline in rescue and reverse studies to year 5 post-treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and nadir to 5 years post-treatment, assessed at baseline in rescue and reverse studies to year 5 post-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
Ocular Adverse Events (AEs)
Secondary outcome measures
Responder Analysis: Clinically Relevant Recovery
Visual Acuity
Other outcome measures
Eyes on Chart

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

RESTORE Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: GS010-treated EyesExperimental Treatment1 Intervention
Lenadogene nolparvovec Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive a single injection of GS010 and the other eye will receive a sham injection. GS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.
Group II: Sham-treated EyesPlacebo Group1 Intervention
Sham Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive GS010 and the other eye will receive a sham injection. Eyes receiving sham injection will undergo the same preparatory procedures as eyes receiving GS010 injection, including pupillary dilation, topical anti-infection and topical anesthetic procedures. Sham intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GS010
2016
Completed Phase 3
~160

Find a Location

Who is running the clinical trial?

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
RESCUE and REVERSE Long-term Follow-up
2018. "RESCUE and REVERSE Long-term Follow-up". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03406104.
~9 spots leftby Apr 2025
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