Genetic clinical trials
What is a clinical trial?
A clinical trial is a type of research study that uses human volunteers. Studies are designed to evaluate the potential benefits, safety, and efficacy of an investigational drug, biologic, or device.
Clinical trials typically go through a period of time when they are accepting new patients to participate. Each trial has a set of guidelines about who can participate, and once the timeframe for enrollment has passed, no new patients can be accepted. To understand more about clinical trials, please review this information from the National Institutes of Health.
Trials currently recruiting participants
Trials listed in this section are currently open for enrollment.
Subretinally injected ushstat
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected UshStat®, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B.
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
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.
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.
On-going studies closed to enrollment
The Casey Ophthalmic Genetics Service is conducting the following trials, but they are no longer enrolling new participants.
X-linked retinoschisis (XLRS)
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.
Oral valproic acid treatment for retinitis pigmentosa
A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Retinitis Pigmentosa Protocol #H-13371
The purpose of this study is to learn about a drug therapy being studied in patients with Retinitis Pigmentosa (RP). Valproic Acid (VPA) has been approved for the treatment of seizures, certain psychiatric disorders, and migraine headaches. The study investigators want to find out if VPA can help people with RP.VPA is taken by mouth.
CNTF: This study is investigating an implant that releases ciliary neurotrophic factor (CNTF), a protein capable of protecting the light-sensitive cells in the eye from further degeneration, in patients with moderate to advanced Retinitis Pigmentosa (RP). Study is closed to enrollment and subjects are currently in follow-up.
CNTO: This study is investigating cell-based therapy in patients with advanced Retinitis Pigmentosa. Study is closed to enrollment and subjects are currently in follow-up.
Leber congenital amaurosis
This study is investigating an experimental gene therapy in patients with LCA caused by mutations (changes) in the RPE65 gene.