- ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
This study is testing a new gene therapy for people with retinitis pigmentosa caused by a specific faulty gene. It's looking at how safe the treatment is and if it helps improve vision. Researchers are giving the treatment into one eye.
France - active not recruiting
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
This study looks at how a specific eye condition, linked to Usher Syndrome 1F and a gene called PCDH15, changes over time. Its goal is to help scientists better measure these changes, which will speed up the development of new treatments for this type of sight loss.
United States · Canada · France - active not recruitingPHASE3
Oral N-acetylcysteine for Retinitis Pigmentosa
This study is testing if a medicine called N-acetylcysteine (NAC) can help slow down vision loss in people with an eye condition called Retinitis Pigmentosa (RP). RP causes changes in your genes that lead to gradual vision loss, especially night vision and side vision.
United States · Austria · Canada - recruiting
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
This study looks at rare genetic eye conditions called retinal dystrophies. It aims to understand how these conditions develop and affect people over time, using genetic information and regular check-ups to improve knowledge and future treatments.
United States · Australia · Belgium - active not recruiting
Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa
This study looks at how Retinitis Pigmentosa (RP) caused by specific gene changes (PDE6A, PDE6B, or RHO) naturally progresses. Researchers want to understand changes in the eye over time to help design better treatments in the future. It doesn't test new medicines, it just observes.
Germany - active not recruitingNA
Prospective Natural History Study of Retinitis Pigmentosa
This study looks at how a particular eye condition called Retinitis Pigmentosa (RP) changes over time. It focuses on people with specific genetic causes of RP to understand how their vision is affected and how the disease progresses.
United States · France - active not recruitingPHASE1, PHASE2
Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation
This study is testing a new cell therapy for an inherited eye condition called retinitis pigmentosa (RP). Doctors are looking at the safety and how well special cells work in the eye to potentially improve or protect vision.
France - active not recruiting
Rate of Progression in EYS Related Retinal Degeneration
This study aims to understand how an eye condition called EYS-related retinitis pigmentosa progresses over time. Researchers want to learn more about how it affects vision to help develop treatments in the future. It involves monitoring people with EYS gene mutations for four years.
United States · Canada · Finland