A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis...
Retinitis PigmentosaThe purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II & III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect one's ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision. The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. In this study, two different CNTF dose levels will be used: a high dose and a low dose in one eye, as well as a sham (or placebo) surgery in the other eye.
Safety Study in Retinal Transplantation for Retinitis Pigmentosa.
Retinitis Pigmentosa.The long-term goal is to show that retinal transplantation can help to prevent blindness and to restore eyesight in patients with the inherited disease retinitis pigmentosa.
Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis...
Retinitis Pigmentosa (RP)This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Four different dose levels of cells will be assessed in each of two groups of patients.
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis...
Retinitis PigmentosaUsher Syndrome Type 21 moreThis clinical trial is a single-site, 30 patient study for participants who have early stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). Funding Source - FDA OOPD and Foundation Fighting Blindness.
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Retinitis PigmentosaThe purpose of this study is to evaluate the short-term safety and efficacy of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal...
Retinitis Pigmentosa (RP)The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65. To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001. To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.
Effectiveness of a Cognitive-behavioral Program of Coping With Psychological Stress in People With...
Vision DisordersStress3 moreThe aim of this study is to evaluate the efficacy of cognitive - behavioral therapy for the control of psychopathological stress and the disease of people with Retinitis Pigmentosa (RP).
The FIGHT-RP1 Study
Retinitis PigmentosaRetinitis Pigmentosa (RP) is a devastating eye disease and at present there are no known treatment options that can alter the rate of vision loss. In a series of studies in animal models, the effects of exposing cones in the periphery of the retina to a large excess of oxygen results in progressive oxidative damage to cone photoreceptors and cone cell death. Compared to control patients, those with RP showed significant reduction in the reduced to oxidized glutathione ratio (GSH/GSSG) in aqueous humor and a significant increase in protein carbonyl content. This demonstration of oxidative stress and oxidative damage in the eyes of patients with RP, suggests that oxidative damage-induced cone cell death in animal models of RP may translate to humans with RP and support the hypotheses that (1) potent antioxidants will promote cone survival and function in patients with RP and (2) aqueous GSH/GSSG ratio and carbonyl content on proteins provide useful biomarkers of disease activity in this patient population. Orally administered N-Acetylcysteine (NAC) has been found to be a particularly effective antioxidant that promotes prolonged cone survival and maintenance of cone function in a mouse model of RP. There is good rationale to test the effect of NAC in patients with RP. The first step is to test different dosing regimens to identify the lowest dose that is able to restore aqueous GSH/GSSG ratio and reduce carbonyl adducts on aqueous proteins. In patients with Idiopathic Pulmonary Fibrosis, polymorphisms within the TOLLIP gene were found to influence outcomes of NAC-treated patients. The product of the TOLLIP gene, toll-interacting protein, is an inhibitory adaptor protein downstream of toll-like receptors, mediators of innate and adaptive immunity. The identification of the influence of TOLLIP polymorphisms on the effect of NAC in Idiopathic Pulmonary Fibrosis provides the rationale for collecting DNA and genotyping the same single nucleotide polymorphisms (SNPs) in the current trial. In addition to this candidate gene genetic analysis, patient RNA will be collected and banked for future transcriptome analysis. The rationale for this is to identify gene expression changes that modify disease progression in RP. There is substantial variability in the rate of progression among patients with RP. A patient who loses all vision early in life can have a sibling with the same mutation who maintains vision into advanced age. This suggests that modifier genes can have a major impact on cone survival. This study will test the hypothesis that the level of expression of gene products that contribute to the antioxidant defense system may influence cone cell death and hence the rate of loss of visual field. It is also possible that gene expression differences may contribute to differences in response to NAC. For these reasons collecting RNA samples from patients will allow next-generation sequencing in the future to understand the transcriptome background on which the study intervention has been performed.
Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis...
Retinitis PigmentosaThis study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients...
Retinitis PigmentosaRetinal Diseases1 moreThe purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)