Clinical trials

At OHSU Casey Eye Institute, we are a leader in gene therapy research, bringing help and hope to those facing sight impairment and blindness. Casey is conducting more gene therapy clinical trials than any other institution in the world and is the first to test this groundbreaking approach for several inherited disorders of the retina. 

Gene therapy is a treatment that involves replacing non-working genes with healthy copies to slow or halt vision loss. Individuals diagnosed with a genetic eye disease may be a candidate for a clinical trial using gene therapy. We enroll study patients from across the United States and throughout the world.

Clinical trials open to enrollment

These studies are actively seeking new participants. Please contact 503-494-0020 or ordc@ohsu.edu if you are interested in participating.

Achromatopsia (CNGB3) Gene Therapy Trial 

The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. This is the first study that aims to treat Achromatopsia disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. 

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Achromatopsia (CNGA3) Gene Therapy Trial 

The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. 

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Choroideremia (CHM) Natural History Study

The purpose of this study is to learn more about Choroideremia; how this condition affects a person’s eyes and vision and add to the knowledge base for this rare disease.

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X-linked Retinitis Pigmentosa (RPGR) Natural History

The purpose of this study is to learn more about a condition called X-Linked Retinitis Pigmentosa (XLRP) caused by changes in the RPGR gene. The study investigators want to find out how changes in the RPGR gene affect vision, and what tests are more helpful for tracking changes in a person's eyes and vision.

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X-linked Retinoschisis (RS1) Gene Therapy Trial

The purpose of this study is to learn about a new gene therapy that may help patients with X-Linked Retinoschisis (XLRS).  This is the first study that aims to treat XLRS disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the vitreous (a thick, gel-like transparent substance that fills the center of the eye) of one eye. The eye with worse vision will receive the gene therapy. 

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Usher Syndrome Type 1B (MYO7A) Gene Therapy Trial

The purpose of this study is to learn about a new gene therapy being studied in patients with Retinitis Pigmentosa (RP) as a result of Usher Syndrome.This is the first study that aims to treat RP due to Usher Syndrome by gene therapy.The study investigators want to find out if UshStat is safe for use in humans.The gene therapy is given by surgical injection underneath the retina of one eye.The eye with worse vision will receive the gene therapy

Stargardt's Macular Degeneration (ABCA4) Gene Therapy Trial

The purpose of this study is to learn about a new gene therapy that may help patients with Stargardt's Macular Degeneration (SMD). This is the first study that aims to treat Stargardt's disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection underneath the retina of one eye. The eye with worse vision will receive the gene therapy. 

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On-going trials closed to enrollment

These studies are ongoing but no longer seeking new participants. 

 Eye imaging technology

Achromatopsia (CNGB3) Natural History Study 

The purpose of this study is to learn about how Achromatopsia, caused by the CNGB3 gene, affects a person's eyes and vision. Specifically the aim for this natural history study is to learn how this condition affects a patient's eyes and vision over a period of time and what tests will be most helpful for tracking vision changes. 

X-linked Retinoschisis (RS1) Natural History Study 

The purpose of this study is to learn more about how to follow changes in vision and other symptoms associated with XLRS. This information is needed for the development of future gene therapy trials. 

Leber Congenital Amaurosis (RPE65) Gene Therapy Trial 

This study is investigating an experimental gene therapy in patients with LCA caused by mutations (changes) in the RPE65 gene. 

Completed Trials 

These studies are completed. 

Oral Valproic Acid Treatment for Retinitis Pigmentosa 

This study investigated the use of oral Valproic Acid (VPA) in participants with autosomal dominant Retinitis Pigmentosa (RP).  

Ciliary Neurotrophic Factor Device Trial 

This study investigated an implant that releases ciliary neurotrophic factor (CNTF), a protein capable of protecting the light-sensitive cells in the eye from further degeneration, in participants with moderate to advanced Retinitis Pigmentosa.  

CNTO 

This study investigated cell-based therapy in participants with advanced Retinitis Pigmentosa.