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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

Study 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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from day 0 to month 12 (12 months)

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

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

Primary outcome measures

Number of Participants Experiencing Adverse Events

Secondary outcome measures

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%

Viral upper respiratory tract infection

17%

Headache

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%

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

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

Do I qualify?Apply below to find out

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

Frequently Asked Questions

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

To what extent is this trial being implemented across various sites?

"Currently, 9 medical sites are enrolling participants for this study; these include Dallas, San Francisco and Boston as well as other nearby centres. To reduce the strain of travelling for patients wishing to join in on the trial, it is best that they select a centre close by them."

Answered by AI

Is this experiment currently accepting participants?

"According to the clinicaltrials.gov details, this trial is not actively seeking participants at present; it was initially posted on May 1st 2015 and has been recently amended July 18th 2022. However, there are two alternative trials that are currently recruiting patients."

Answered by AI

Recent research and studies

Progress in Retinal and Eye ResearchJournal

X-linked Juvenile Retinoschisis: Clinical Diagnosis, Genetic Analysis, and Molecular Mechanisms

Molday, Robert S., Ulrich Kellner, and Bernhard H.F. Weber. 2012. “X-linked Juvenile Retinoschisis: Clinical Diagnosis, Genetic Analysis, and Molecular Mechanisms”. Progress in Retinal and Eye Research. Elsevier BV. doi:10.1016/j.preteyeres.2011.12.002.

clinicaltrials.govStudy

Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)

2015. "Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02416622.