pompe-disease Clinical Trials
A listing of pompe-disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 57 clinical trials
Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies
Summary of the Study This clinical trial evaluates a novel peptide-based therapy for treating retinal dystrophies, age-related macular degeneration (AMD), and diabetic retinopathy (DR). The therapy consists of peptides derived from fetal tissues, mesenchymal stem cells (MSCs), and bioactive growth factors, administered sublingually for systemic absorption. Study Objectives: Primary Objectives: …
Multicenter Clinical Study of the SING-IMT in Patients with Late-stage AMD
The objective of this study is to determine the safety and effectiveness of the smaller incision, new generation (SING), implantable miniature telescope (IMT) in patients with moderate-severe central vision loss due to late-stage age-related macular degeneration (AMD).
Survival of Monocytes Collected From Patients With Atrophic AMD in Retinal Pigmented Epithelium Explants
Age-related macular degeneration (AMD) affects 2 million people in France and is the main cause of irreversible blindness in France. All patients initially have an early form of the disease. This early form can evolve in two different ways: the atrophic form, which progresses slowly, and the exudative or neovascular …
Dark Adaptation as an Early Indicator of Response to Statin Therapy for Intermediate AMD
interventional trial for off label use of high dose atorvastatin 80 mg in intermediate AMD patients and correlate recovery response measured by dark adaptation recovery time with drusen volume reduction measured by SD-OCT
Enhanced Vision and Imaging Tests for Enabling Treatment Trials in Early and Intermediate AMD
This monocentric prospective longitudinal observational study will validate fundus-tracked dark adaptometry as an endpoint for future treatment trials in early and intermediate age-related macular degeneration (AMD). The study will characterize normative cone- and rod-mediated dark adaptation parameters in healthy volunteers, assess test-retest reliability, quantify sensitivity to change over time, evaluate …
Multicenter Real-life Observational Study Switched Aflibercept or Ranibizumab to Faricimab in Patients With AMD.
Age-related macular degeneration (AMD) is a degenerative retinal disease. The prognosis of the exudative form was transformed by the introduction of the anti-VEGF monoclonal antibody treatments ranibizumab \[1\] and aflibercept \[2\] in the 2010s. In 2022, a new molecule, Faricimab, proved its efficacy in exudative AMD. It is a bi-specific …
Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD
The purpose of this study is to collect efficacy and safety information of intravitreal injection for 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies in patients with GA associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability …
The Role of Occupational Therapy for Improving Medication Management for Persons With AMD, DR and Glaucoma
The goal of this clinical trial is to learn if education about additional task lighting, enhanced contrast, enlarged print instructions, smart apps and organizational strategies will improve medication management skills. The main questions it aims to answer are: Does additional task lighting, enhanced contrast, enlarged print instructions, smart apps and …
A Phase IIb Randomized, Multicenter Trial of Subretinal CPCB-RPE1 in Advanced Dry AMD (Geographic Atrophy)
This is a Phase IIb randomized, clinical trial designed to assess the safety and efficacy of unilateral implantation of the CPCB-RPE1 implant in subjects with geographic atrophy involving the fovea. Up to 6 surgical implantation sites will deliver the CPCB-RPE1 in this Phase IIb clinical trial. Additional study sites may …
Detection and Management of Sinusitis in a Single-center Prospective Cohort of Patients With Exudative AMD (SINE)
An inflammatory component associated with AMD has been highlighted by genetic associations of predisposition to AMD, as well as by the recently demonstrated link between AMD and periodontitis. Some patients followed at the Fondation Adolphe de Rothschild Hospital for wet AMD seemed to show an improvement of neovascular activity (less …
