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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 National Eye Institute (NEI)

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up months 1, 2, 3, 4, 6, 9, 18 and annually at years 2-5

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ months 1, 2, 3, 4, 6, 9, 18 and annually at years 2-5

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Occurrence of AEs

Ocular Structure

Retinal function

Secondary outcome measures

Anti-AAV antibodies

ERG

OCT imaging

+1 more

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

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Eye Institute (NEI)Lead Sponsor

546 Previous Clinical Trials

1,401,916 Total Patients Enrolled

Henry E Wiley, M.D.Principal InvestigatorNational Eye Institute (NEI)

5 Previous Clinical Trials

179 Total Patients Enrolled

Catherine A Cukras, M.D.Principal InvestigatorNational Eye Institute (NEI)

11 Previous Clinical Trials

1,555 Total Patients Enrolled

Media Library

RS1 AAV Vector (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02317887 — Phase 1 & 2

Inheritance Research Study Groups: Group 3, Group 4, Group 1, Group 5, Group 6, Group 2

Inheritance 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

Frequently Asked Questions

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

What is the intended outcome of this research endeavor?

"The primary purpose of this lengthy trial, which includes evaluations on Day 1, Seven days later, two weeks after that and then at Month 1, 2 and 3 before having additional check-ups every three months until Year 5 is to assess the ocular structure. Secondary objectives are tracking Anti-AAV antibodies formation in serum samples as well as changes in ERG response amplitudes and OCT imaging compared with baseline results."

Answered by AI

Are there any slots still available for participants in this trial?

"Confirmed, the clinical trials website states that this research is presently not recruiting new participants. First posted on February 11th 2015 and last updated June 3rd 2022, it appears to be inactive at this time but there are two other active studies looking for volunteers."

Answered by AI

Recent research and studies

Molecular TherapyJournal

Retinal AAV8-RS1 Gene Therapy for X-linked Retinoschisis: Initial Findings from a Phase I/iia Trial by Intravitreal Delivery

Cukras, Catherine, Henry E. Wiley, Brett G. Jeffrey, H. Nida Sen, Amy Turriff, Yong Zeng, Camasamudram Vijayasarathy, et al.. 2018. “Retinal AAV8-RS1 Gene Therapy for X-linked Retinoschisis: Initial Findings from a Phase I/iia Trial by Intravitreal Delivery”. Molecular Therapy. Elsevier BV. doi:10.1016/j.ymthe.2018.05.025.

Molecular TherapyJournal

Immune Function in X-linked Retinoschisis Subjects in an AAV8-RS1 Phase I/iia Gene Therapy Trial

Mishra, Alaknanda, Camasamudram Vijayasarathy, Catherine A. Cukras, Henry E. Wiley, H. Nida Sen, Yong Zeng, Lisa L. Wei, and Paul A. Sieving. 2021. “Immune Function in X-linked Retinoschisis Subjects in an AAV8-RS1 Phase I/iia Gene Therapy Trial”. Molecular Therapy. Elsevier BV. doi:10.1016/j.ymthe.2021.02.013.

Progress in Retinal and Eye ResearchJournal

Of Men and Mice: Human X-linked Retinoschisis and Fidelity in Mouse Modeling

Vijayasarathy, Camasamudram, Sheik Pran Babu Sardar Pasha, and Paul A. Sieving. 2022. “Of Men and Mice: Human X-linked Retinoschisis and Fidelity in Mouse Modeling”. Progress in Retinal and Eye Research. Elsevier BV. doi:10.1016/j.preteyeres.2021.100999.

clinicaltrials.govStudy