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 48 clinical trials
Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
Gene Therapy(FT-003) for Wet AMD
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of disposable vitreous injection of FT-003 in subjects with neovascular age-related Macular Degeneration (nAMD). The study was divided into two phases, Phase 1 dose escalation and Phase II dose expansion.
Active AMD Study to Improve Function in Veterans
Age Related Macular Degeneration (AMD) is the leading cause of vision loss among Veterans aged 50 years and older. AMD also adversely affects mortality, physical and cognitive functioning, and activities of daily living. These debilitations negatively impact quality of life for US Veterans. Unfortunately, there are no cures and few …
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement. Conducted at the Centre de Référence Liégeois des Maladies Neuromusculaires in Liège, Belgium, the study will enroll 300 ambulant patients, including individuals with neuromuscular disorders …
QA108 Phase III Study in Subjects With Intermediate AMD
This is a phase III randomized, double-masked, placebo-controlled, multicenter study. To evaluate the efficacy and safety of QA108 granules in the treatment of intermediate age-related macular degeneration.
Progression Suppression and Retinal Regression in VEGF-resistant AMD
The goal of this clinical trial is to assess safety and efficacy in patients with Neovascular Age-related Macular Degeneration with no response to existing therapy. The main measures it aims to answer are: Investigation of adverse events Changes in clinical testing data Changes in vital signs Changes in intraocular pressure …
AMD and Validation Rod-Meditated Dark Adaptation With Everyday Task Performance
The goal is to conduct a cross-sectional, single timepoint study on older adults with early and intermediate AMD, and with subretinal drusenoid deposits (SDD), and those in normal health, establishing an association between dark adaptation and reading performance under dim illumination, both which depend on rod photoreceptors.
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).
Evaluation of Therapeutic Efficacy of Faricimab for Clinical AMD, DME, and RVO Patients
The goal of this clinical trial is to learn if drug Faricimab works to treat nAMD, DME or RVO in adults. It will also learn about the safety of drug Faricimab. The main questions it aims to answer are: Does drug Faricimab can improve the best-corrected visual acuity of participants? …
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: …
