Ophthalmic genetics clinical trials

An important part of the Ophthalmic Genetics Service's mission is to conduct research that will lead to effective treatments for inherited retinal degenerations.

Ongoing 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.

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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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.

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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

Eye imaging technology

These studies are ongoing but no longer seeking new participants.

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.