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A clinical trial evaluating the safety and Efficacy of a single subretinal injection of AGTC-501 in participants with X-linked retinitis pigmentosa caused by RPGR mutations.
Overview
This research project is testing a new treatment for X-linked Retinitis Pigmentosa (XLRP). The new treatment is called AGTC-501.
XLRP is a genetic (inherited) eye disease that affects cells in the retina, the layer at the back of the eye that detects light. XLRP results in loss of vision due to degeneration (breakdown) of special cells in the retina called photoreceptors, which are needed for vision. This causes night blindness and gradual worsening of your vision.
A gene that is needed for normal vision is called Retinitis Pigmentosa GTPase Regulator (RPGR). XLRP can be caused by a change (mutation) in this gene.
The purpose of this Phase 2/3 study is to see if the investigational study drug, AGTC-501, works to preserve and/or improve vision and other symptoms of XLRP, compared to participants in this study who will not be treated with AGTC-501 in at least the first year.
The study drug contains a healthy copy of the RPGR gene and parts of a virus called adeno- associated virus (AAV). The AAV virus does not cause known disease or illness in humans. The AAV works like a delivery system to get healthy RPGR gene copies into your cells after injecting the study drug into your eye. This experimental procedure is called gene transfer.
If you are suitable and agree to take part in this study, participation will last up to 6 years and you will be required to return to the study site for 18 study visits.
- Principal Investigator
Dr Tom Edwards
This research project is testing a new treatment for X-linked Retinitis Pigmentosa (XLRP). The new treatment is called AGTC-501.
XLRP is a genetic (inherited) eye disease that affects cells in the retina, the layer at the back of the eye that detects light. XLRP results in loss of vision due to degeneration (breakdown) of special cells in the retina called photoreceptors, which are needed for vision. This causes night blindness and gradual worsening of your vision.
A gene that is needed for normal vision is called Retinitis Pigmentosa GTPase Regulator (RPGR). XLRP can be caused by a change (mutation) in this gene.
The purpose of this Phase 2/3 study is to see if the investigational study drug, AGTC-501, works to preserve and/or improve vision and other symptoms of XLRP, compared to participants in this study who will not be treated with AGTC-501 in at least the first year.
The study drug contains a healthy copy of the RPGR gene and parts of a virus called adeno- associated virus (AAV). The AAV virus does not cause known disease or illness in humans. The AAV works like a delivery system to get healthy RPGR gene copies into your cells after injecting the study drug into your eye. This experimental procedure is called gene transfer.
If you are suitable and agree to take part in this study, participation will last up to 6 years and you will be required to return to the study site for 18 study visits.
- Principal Investigator
Learn more
View this study on ClinicalTrials.gov
See the ClinicalTrials.gov database listing for more detailed information about this study.