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NCT02416622 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of X-linked Retinoschisis, sponsored by Beacon Therapeutics.

Completed
Registry status
Phase 1
Development phase
27
Enrollment target
9
Study locations

NCT02416622: Completed Phase 1 study of X-linked Retinoschisis, sponsored by Beacon Therapeutics.

NCT02416622 is a Phase 1 study of X-linked Retinoschisis that has completed, run by Beacon Therapeutics. The registered enrollment target is 27 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (55% lower). The trial reports 9 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02416622, a Phase 1 study of X-linked Retinoschisis, has completed, sponsored by Beacon Therapeutics.

COMPLETED
Registry status
Phase 1
Development phase
27 participants
Enrollment target
9
Study locations

Study 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.

Primary Outcome

Number of participants with ocular adverse events, assessed by standard ophthalmic examination, including slit lamp biomicroscopy, tonometry, and indirect ophthalmoscopy.

Conditions Studied

Interventions

  • BIOLOGICAL rAAV2tYF-CB-hRS1

Study Locations (9)

Texas

  • Retina Foundation of the Southwest - Dallas
  • Baylor College of Medicine, Alkek Eye Center - Houston

California

  • University of California San Francisco, Dept. of Ophthalmology - San Francisco

Florida

  • University of Miami - Miller School of Medicine Bascom Palmer Eye Institute - Miami

Maryland

  • The Wilmer Eye Institute, Johns Hopkins Hospital - Baltimore

Massachusetts

  • Massachusetts Eye and Ear Infirmary - Boston

Michigan

  • University of Michigan Kellogg Eye Center - Ann Arbor

North Carolina

  • Duke Eye Center, Duke University Medical Center - Durham

Oregon

  • Casey Eye Institute, Oregon Health and Sciences University - Portland

Trial Details

FieldValue
Enrollment Target 27 participants
Start Date 2015-05
Est. Completion 2023-05-09
Phase Phase 1
Beacon Therapeutics

12 total trials

What the finished NCT02416622 record still lists

NCT02416622 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (55% lower).

The record links to 1 condition, with X-linked Retinoschisis appearing as the primary indexed condition, and to 1 intervention - of which rAAV2tYF-CB-hRS1 is the first listed.

NCT02416622 lists 9 locations in 8 states (Texas, California, Florida).

Frequently Asked Questions

What is clinical trial NCT02416622 about?

NCT02416622 is a clinical study titled "Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)". 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.

What is the current status of trial NCT02416622?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 27 participants. The study started on 2015-05. Estimated completion is 2023-05-09.

What conditions does trial NCT02416622 study?

This clinical trial studies the following conditions: X-linked Retinoschisis.

What interventions are being tested in trial NCT02416622?

The interventions under investigation include: rAAV2tYF-CB-hRS1 (BIOLOGICAL).

Who is sponsoring clinical trial NCT02416622?

This trial is sponsored by Beacon Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT02416622 being conducted?

This trial has 9 study locations across California, Florida, Maryland, Massachusetts, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for X-linked Retinoschisis

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT02416622's enrollment target sits among peer trials

27 1463rd of 2000 higher than 519 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02416622, the US trial registry maintained by the National Library of Medicine. NCT02416622 (small enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.