View clinical trials related to Stargardt Disease.
Filter by:Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease.
Oral disulfiram (Antabuse®) has been shown to improve image-forming vision in animal models with retinal degeneration due to its ability to decrease Retinoic Acid synthesis and consequently reduce hyperactivity in the inner retina. The investigator will aim to evaluate the impact of oral disulfiram on the vision of patients with retinal degeneration who are being treated with the drug in the management of their concurrent alcohol use disorder.
The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1). JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene.
The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease. This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 42 subjects.
Patients who develop macular diseases have several clinical complications,such as central vision loss, the central scotoma of the visual field, the decrease of reading speed and fixation stability. At present, there is still no satisfactory effect in the prevention and treatment of advanced macular disease. A new rehabitation strategy named microperimetric biofeedback training has been shown to be effective in improving patients' visual appearance, but there is no consensus regarding the optimal methodology and standard of practice. Therefore, we designed a prospective clinical study to verify the effectiveness of MBFT and to determine an optimal plan.
Some phenotypes of Stargardt disease are rather distinct. This includes the 'bull's eye maculopathy' phenotype associated with the frequent ABCA4 G1961E variant. In anticipation of a treatment trial, this natural history study aims to compare functional and structural outcome measures systematically.
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis. This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.
Rare ocular diseases (ROD) are a heterogeneous group of ocular diseases that affect very few people and, generally, for which no tretament is available. An important subgroup of these diseases are inherited retinal degenerations. In this study we focus on understanding the natural history of different ROD that affect the posterior segment.