Gene therapy watch: 5-Year safety check for inherited blindness treatment
NCT ID NCT04312672
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study followed 42 people who received a gene therapy for X-linked retinitis pigmentosa, an inherited eye disease that causes blindness. The goal was to check the treatment's safety over up to 5 years. Researchers also looked at whether patients' vision and ability to move around in low light improved.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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42 people
The number who actually took part.
- Started
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Jul 2017
- Finished
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Sep 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Males receiving AAV5-hRKp.RPGR in the MGT009 study
- Ages
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5 years and older
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Received AAV5-hRKp.RPGR in the MGT009 Study Exclusion Criteria: * Unwilling or unable to meet with the requirements of the study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF, United Kingdom
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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, EC1V 9EL, United Kingdom
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Univ of Michigan Medical Center
Ann Arbor, Michigan, 48105, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Gene therapy offers hope for rare Blindness-Causing eye disease
- Gene therapy targets second eye in rare blindness disorder
- Gene therapy offers hope for rare blindness
- Experimental gene therapy aims to halt vision loss in rare eye disease
- Gene therapy shows promise for rare blindness disease