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

Completed · Phase 1/Phase 2

Conditions studied: X-linked Retinoschisis

In brief

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.

Key facts

Study ID
NCT02416622
Run by
Beacon Therapeutics
People needed
27
Starts
2015-05-01
Expected to finish
2023-05-09
Last updated by the study team
2023-06-12

Who can join

Age: 6 and older. Sex: male. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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