Active clinical trials for "Blindness"

A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Five cohorts will be included in this trial. Cohorts 1, 2 and 4 will consist of individuals 18 years of age and older. Cohorts 3 and 5 will consist of individuals between the ages of 8 and 17, inclusive. Enrollment in Cohorts 3 and 5 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older groups of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention. The goal of this clinical trial is to determine the safety of uniocular subretinal administration of rAAV2-CBSB-hRPE65 in individuals with RPE65-associated retinal disease. Ocular and systemic toxicity will be assessed prior to and following vector administration to determine if there are adverse changes that may be associated with vector administration.

The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.

The study is a follow-on to a Phase 1 dose-escalation and safety study.

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

This is a pilot study of a patient navigator intervention for people living with diabetes and at high risk of diabetic blindness. The investigators are assessing the feasibility and acceptability of the intervention in preparation for a clinical trial.

In this study, the principle functionality of the device will be tested in humans for the first time. The study will evaluate the extent to which patients with atrophic dry age related macular degeneration (AMD) have evoked light perception using the implant.

Blindness and visual impairment severely impact the visual health and life quality of people, particularly the 2.566 million senior citizens aged at 65 and above in Shanghai. The main reason is uncorrected refractive error, of which, 62.1% can be solved through refractive correction. For this reason, the uncorrected refractive errors of 154,000 senior citizens in Shanghai can be taken as a priority among the public health issues to prevent blindness. Now, with the aim to reduce the prevalence rate of blindness and visual impairment, it is planned to establish a public health service mechanism in terms of refractive error screening and correction for the elderly by relying on Shanghai's three-level (city-district-community) eye diseases prevention network, using proper refractive correction technology, and moving related services forward to communities in order to screen, identity, and correct blindness and visual impairment caused by refractive errors as early as possible.

To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).

This study seeks to determine the extent of the visual capabilities that can be restored in hemianopic stroke patients by a multisensory training technique and evaluate changes in the brain that the training induces. The effectiveness of the technique will be evaluated in two interventional contexts: patients whose blindness is long-standing and stable, and another in which intervention is as soon as possible after the stroke.