Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Completed

Conditions studied: X-Linked Retinitis Pigmentosa

In brief

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Key facts

Study ID

NCT04312672

Run by

Janssen Research & Development, LLC

People needed

42

Starts

2017-07-31

Expected to finish

2025-09-17

Last updated by the study team

2026-01-21

Who can join

Age: 5 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Received AAV5-hRKp.RPGR in the MGT009 Study

You may not qualify if…

• Unwilling or unable to meet with the requirements of the study

Where it is running

• Massachusetts Eye and Ear Infirmary — Boston, Massachusetts, United States
• Univ of Michigan Medical Center — Ann Arbor, Michigan, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Leeds Teaching Hospitals NHS Trust — Leeds, United Kingdom
• Moorfields Eye Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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