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
Project AMD: Comprehensive Characterisation of Age-Related Macular Degeneration and Its Progression
Age-related macular degeneration (AMD) is the leading cause of irreversible vision loss worldwide, and nearly two million Australians have some signs of AMD. This proposed project is a prospective, observational study that seeks to to understand the underlying aetiology of AMD, factors associated with differences between age-related macular degeneration (AMD) …
Real-World Evaluation of AI Enabled Multi-Spectral Imaging (MSI) for AMD Biomarker Detection
The goal of this observational clinical study is to learn if DeepMSI AI detects age-related macular degeneration (AMD) biomarkers with sensitivity and specificity equivalent to experienced clinicians in adults over 40 years old. The main questions it aims to answer are: Does DeepMSI AI detect AMD biomarkers with sensitivity equivalent …
Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants With Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 Study
Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants with Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 Study
Predicting Risk of Progression of Early to Late AMD in the Aging Eye Through Imaging and Multimodal Evaluation
The purpose of this research study is to evaluate a new screening procedure for the early detection and monitoring of Age-related Macular Degeneration (AMD) in community settings and community hospital settings, to identify associated risk factors, and develop personalized monitoring strategies for at-risk individuals throughout the 5 years follow up …
A Phase 2 Efficacy and Safety Study of GAL-101, 2% Ophthalmic Solution in Non-foveal Geographic Atrophy Secondary to Non-neovascular AMD
Age-related macular degeneration (AMD) affects millions of elderly patients. When advanced, there is Geographic Atrophy (GA) in the retina. This means that there is an area with a loss of light-sensitive cells, called photoreceptors. That part of the retina can no longer see. Atrophy begins as a small spot in …
Medico-economic Evaluation of Anti-VEGF Treatments in the Treatment of naïve Age-related Macular Degeneration (AMD): a Model Adapted to the French Context
Age-related macular degeneration (AMD) is the leading cause of blindness after 50 years in industrialized countries, compromising daily activities and reducing quality of life. And whose treatment is one of the first health expenditures. Since September 2015, Bevacizumab, which does not have marketing authorization in AMD, has been given a …
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.
A Long-term Study for the Outcome of Pompe Disease
Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have …
Cognitive and Neurological Pathologies in Pompe Disease
The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. …
