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 45 clinical trials
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
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 …
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 …
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 Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
The proposed clinical investigation wants primary to validate the safety of the innovative light therapy approach and in second priority provide insight and confirmations on therapeutic effect. By combining two clinically standard laser-light treatment, both exhibiting a solid-safe profile: the photothermal and the photobiological techniques; the investigational device (reSEES) wants …
Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.
Atrophic Age-related Macular Degeneration (AMD) Treated With Intravitreal Injections of Umbilical Cord Blood Platelet-rich Plasma (CB-PRP): a Pilot Study
The objective of the study will be to evaluate the efficacy of intravitreal injections of Umbilical Cord Blood Platelet-rich Plasma (CB-PRP) in order to reduce or stabilize the atrophic progression in dry Age-related Macular Degeneration (AMD)
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 …
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01 in Patients With Geographic Atrophy Secondary to Dry AMD
This is a Phase 1/2 study, multi-center, dose-escalation interventional study of BS01 in subjects with GA secondary to dry AMD. Part 1 is an open label dose-escalation study; Part 2 is a dose-expansion study with dose(s) selected from Part 1 based on a benefit/risk assessment, and an untreated (sham injection) …
A Study to Investigate Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity of RO7669330 in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
The main purpose of this study is to assess the ocular and systemic safety and tolerability of RO7669330 in GA secondary to AMD after multiple unilateral intravitreal (IVT) doses.
