CompletedPhase 1/2

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

United States27 participantsStarted 2015-05

Plain-language summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format.

Who can participate

Age range

6 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene
. Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase),
. Able to perform tests of visual and retinal function,
. Visual acuity specified for each group
. Not treated with CAIs currently or within 3 months prior to study enrollment,
. Have acceptable laboratory parameters.

Exclusion criteria

. Prior receipt of any AAV gene therapy product,
. Pre-existing eye conditions that would preclude the planned intravitreal injection or interfere with interpretation of study endpoints or complications of vector administration.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants Experiencing Adverse Events

Timeframe: From Day 0 to Month 12 (12 months)

Trial details

NCT IDNCT02416622

SponsorBeacon Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-04

Results submitted2020-05-18

View on ClinicalTrials.gov More X-linked Retinoschisis trials