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Virus Therapy

rAAV2tYF-CB-hRS1 for Retinoschisis

Phase 1 & 2
Waitlist Available
Research Sponsored by Applied Genetic Technologies Corp
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 from day 0 to month 12 (12 months)
Awards & highlights
No Placebo-Only Group

Summary

This trial will test a new treatment for X-linked retinoschisis, a degenerative eye disease. Up to 27 patients will receive different doses of the treatment to see if it is safe and effective.

Eligible Conditions
  • X-linked Retinoschisis
  • Retinoschisis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from day 0 to month 12 (12 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and from day 0 to month 12 (12 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants Experiencing Adverse Events
Secondary study objectives
Change From Baseline in B-wave Amplitude in Electroretinogram (ERG) Responses
Change From Baseline in Best Corrected Visual Acuity (BCVA)
Change From Baseline in Schisis Cavity Size on Optical Coherence Tomography (OCT)

Side effects data

From 2023 Phase 1 & 2 trial • 27 Patients • NCT02416622
50%
Vitritis
33%
Conjunctival haemorrhage
33%
Keratic precipitates
33%
Anterior chamber inflammation
17%
Headache
17%
Viral upper respiratory tract infection
17%
Atrial Fibrillation
17%
Faecaloma
17%
Pulmonary embolism
17%
Eye pain
17%
Vitreal cells
17%
Conjunctival hyperaemia
17%
Eyelid ptosis
17%
Injection site haemorrhage
17%
Intraocular pressure
17%
Nausea
17%
Increased appetite
17%
Cerebellar stroke
17%
Back pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
rAAV2tYF-CB-hRS1 1 x 10^11 vg Per Eye
rAAV2tYF-CB-hRS1 3 x 10^11 vg Per Eye
rAAV2tYF-CB-hRS1 6 x 10^11 vg Per Eye

Awards & Highlights

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

Trial Design

4Treatment groups
Experimental Treatment
Group I: Groups 2 and 2AExperimental Treatment1 Intervention
Subjects at least 6 y/o treated with a middle dose of rAAV2tYF-CB-hRS1 study drug.
Group II: Groups 1A and 1BExperimental Treatment1 Intervention
Subjects at least 18 y/o treated with a lower dose of rAAV2tYF-CB-hRS1 study drug.
Group III: Group 4Experimental Treatment1 Intervention
Subjects at least 6 y/o treated with a maximum tolerated dose of rAAV2tYF-CB-hRS1 study drug determined for Groups 1A, 1B, 2 and 3.
Group IV: Group 3Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with a higher dose of rAAV2tYF-CB-hRS1 study drug.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
rAAV2tYF-CB-hRS1
2015
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

Applied Genetic Technologies CorpLead Sponsor
10 Previous Clinical Trials
326 Total Patients Enrolled
1 Trials studying Retinoschisis
66 Patients Enrolled for Retinoschisis
Theresa Heah, MDStudy DirectorApplied Genetics Technologies Corporation
7 Previous Clinical Trials
2,289 Total Patients Enrolled
~3 spots leftby Nov 2025
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