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Virus Therapy

Gene Therapy for Retinoschisis

Phase 1 & 2
Waitlist Available
Led By Henry E Wiley, M.D.
Research Sponsored by VegaVect, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has at least one eye that meets the study eye criteria
Participant must agree not to receive live (attenuated) vaccines prior to dosing and for some duration following dosing
Must not have
Participant has compromised renal function
Participant has pre-existing ocular tumors
Timeline
Screening 3 weeks
Treatment Varies
Follow Up months 1, 2, 3, 4, 6, 9, 18 and annually at years 2-5
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a gene transfer vector (AAV-RS1) to see if it is safe to use in people with XLRS.

Who is the study for?
Adults over 18 with X-linked juvenile retinoschisis (XLRS), a specific eye condition, and vision of 20/63 or worse in one eye. Participants must have an RS1 gene mutation confirmed by genetic testing, be able to follow the study protocol and agree to use effective contraception.
What is being tested?
The trial is testing the safety of a new treatment involving injecting a healthy RS1 gene into the eye using a virus vector called AAV-RS1. The goal is to see if this can help produce healthy retinoschisin protein for better retina function.
What are the potential side effects?
Potential side effects are not detailed but will be monitored throughout multiple visits post-injection. Side effects could relate to immune reactions due to the viral vector or local effects from the injection procedure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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One of my eyes meets the study's requirements.
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I agree not to get live vaccines before and after receiving the treatment.
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I am a male with an RS1 gene mutation.
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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My kidney function is not normal.
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I have tumors in my eyes.
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I am on medication that conflicts with the study drugs.
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My study eye had surgery less than 6 months ago.
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My study eye has clear issues that prevent proper retina examination.
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My study eye has other conditions that could cause significant vision loss.
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My high blood pressure is not under control.
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I have liver disease with high liver enzyme levels.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~months 1, 2, 3, 4, 6, 9, 18 and annually at years 2-5
This trial's timeline: 3 weeks for screening, Varies for treatment, and months 1, 2, 3, 4, 6, 9, 18 and annually at years 2-5 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Occurrence of AEs
Ocular Structure
Retinal function
Secondary study objectives
Anti-AAV antibodies
ERG
OCT imaging
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

6Treatment groups
Experimental Treatment
Group I: Group 6Experimental Treatment1 Intervention
Not to exceed 6e11 vg/eye
Group II: Group 5Experimental Treatment1 Intervention
Not to exceed 3e11 vg/eye
Group III: Group 4Experimental Treatment1 Intervention
1e11 vg/eye
Group IV: Group 3Experimental Treatment1 Intervention
1e11 vg/eye
Group V: Group 2Experimental Treatment1 Intervention
1e10 vg/eye
Group VI: Group 1Experimental Treatment1 Intervention
1e9 vg/eye

Find a Location

Who is running the clinical trial?

VegaVect, Inc.Lead Sponsor
National Eye Institute (NEI)NIH
555 Previous Clinical Trials
1,407,380 Total Patients Enrolled
3 Trials studying Retinoschisis
376 Patients Enrolled for Retinoschisis
Henry E Wiley, M.D.Principal InvestigatorNational Eye Institute (NEI)
5 Previous Clinical Trials
179 Total Patients Enrolled

Media Library

RS1 AAV Vector (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02317887 — Phase 1 & 2
Retinoschisis Research Study Groups: Group 3, Group 4, Group 1, Group 5, Group 6, Group 2
Retinoschisis Clinical Trial 2023: RS1 AAV Vector Highlights & Side Effects. Trial Name: NCT02317887 — Phase 1 & 2
RS1 AAV Vector (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02317887 — Phase 1 & 2
~1 spots leftby Jul 2025
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