Promising ROd-cone DYstrophy Gene therapY
Retinitis PigmentosaThis is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm controlled double-masked randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene.
Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II
Retinitis PigmentosaThe study will perform UC-MSCs and CM transplantation. There are two groups with different dosages. The first group will be transplanted with 1.5 million cells, meanwhile, the second group is 5 million cells. Each group consists of 30 subjects. All groups will be transplanted via the peribulbar route. All groups will be observed until six months.
Pilot Study of AuTNA I
Retinitis PigmentosaThe objective of the study is to evaluate: Safety of AuTNA I for subretinal implantation in patients with retinitis pigmentosa; Efficacy of AuTNA I for subretinal implantation in patients with retinitis pigmentosa.
The Effect of Stem Cells and Stem Cell Exosomes on Visual Functions in Patients With Retinitis Pigmentosa...
Retinitis PigmentosaThis clinical trial aims to study the efficacy of umbilical cord-derived mesenchymal stem cells and their exosomes in the treatment of retinitis pigmentosa. The participants will be randomized into 3 groups. Functional and structural parameters will be compared before and after the injections and also will be compared among the groups to reveal whether stem cell and their exosomes are more effective than placebo.
Safety and Tolerability Study of Gene Editing Drug ZVS203e in Participants With Retinitis Pigmentosa...
Retinitis PigmentosaThe purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of ZVS203e administered via subretinal injection in participants with RP caused by RHO site-specific gene mutation (RHO-RP).
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital...
Retinitis PigmentosaLeber Congenital AmaurosisThis is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101). To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study (OCU400-104)i This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study.
BS01 in Patients With Retinitis Pigmentosa
Retinitis PigmentosaNon-randomized, open label, Phase 1/2 dose escalation study of BS01, a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP).
Stem Cell Ophthalmology Treatment Study II
Retinal DiseaseAge-Related Macular Degeneration17 moreThis study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
X-Linked Retinitis PigmentosaA clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
Oral N-acetylcysteine for Retinitis Pigmentosa
Retinitis PigmentosaRetinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several mistakes in the genetic code. Such mistakes are called mutations. The mutations cause degeneration of rod photoreceptors which are responsible for vision in dim illumination resulting in night blindness. After rod photoreceptors are eliminated, gradual degeneration of cone photoreceptors occurs resulting in gradual constriction of side vision that eventually causes tunnel vision. Oxidative stress contributes to cone degeneration. N-acetylcysteine (NAC) reduces oxidative stress and in animal models of RP it slowed cone degeneration. In a phase I clinical trial in patients with RP, NAC taken by month for 6 months caused some small improvements in two different vision tests suggesting that long-term administration of NAC might slow cone degeneration in RP. NAC Attack is a clinical trial being conducted at many institutions in the US, Canada, Mexico, and Europe designed to determine if taking NAC for several years provides benefit in patients with RP.