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 55 clinical trials
Pharmacogenomics of antiVEGF in Patients With Age-Associated Macular Degeneration (AMD)
This is a phase III, multicenter, randomized double-blinded clinical trial with two parallel groups (ranibizumab and bevacizumab) and an observational follow-up of patients who meet elegibility criteria and decline participation due to treatment randomization. It will be performed involving 630 eyes from patients with wet age-related macular degeneration (wAMD) diagnosis …
Natural History of Pompe Disease
The project is a prospective study in which patients affected by adult-onset Pompe disease with c.-32-13T\>G mutation in the GAA gene will be followed-up during two years to describe the natural history using clinical, imaging, histological and molecular parameters. Secondary objectives are: To identify biomarkers for assessing efficacy of future …
Treatment of Neovascular AMD: Artificial Intelligence in Real-world Setting
The purpose of this study is to implement quantitative assessment tools for the treatment of active neovascular AMD patients in a real-world setting in order to provide advantages for both patients (treatment burden) and healthcare system (scheduling visits/treatments).
Ethical Issues of Using Gene Therapy in the Treatment of AMD
The objective of this study is to examine the legitimacy of gene therapy use in the treatment of exudative age-related macular degeneration (AMD). This involves questioning its beneficence/non-maleficence ratio given the limited experience with this innovative technology and its irreversible effects, requiring a surgical procedure for administration. Additionally, the study …
Pompe Pregnancy Sub-Registry
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which …
Pompe Disease Registry Protocol
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, …
A Global Prospective Observational Registry of Patients With Pompe Disease
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: To …
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
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 …
