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.

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Found 37 clinical trials
Treat-and-extend Using Aflibercept for Type 3 Neovascularization

Type 3 neovascularization is a subtype of neovascular age-related macular degeneration (AMD) that is characterized by intraretinal neovascularization. Treat-and-extend (TAE) regimen is a widely

  • 0 views
  • 22 Sep, 2022
  • 1 location
A Clinical Study to Evaluate the Safety and Feasibility of the OcuDyne System in the Treatment of Age-Related Macular Degeneration (AMD)

This study evaluates the safety and feasibility of using the OcuDyne OPTiC System in patients with late-stage non-exudative age-related macular degeneration.

  • 0 views
  • 21 Sep, 2022
  • 1 location
A Phase 2, Multicenter, Prospective, Randomized, Double-Masked, Parallel Study of EYP-1901, a Tyrosine Kinase Inhibitor (TKI), Compared to Aflibercept in Subjects With Wet AMD

This is a phase 2 randomized, double -masked study comparing the efficacy of EYP-1901 at two dose levels: 2060 ug and 3090 ug against Aflibercept.

  • 0 views
  • 16 Sep, 2022
Povidone Iodine Efficacy Study (PIES)

This is a single center, randomized, double-blind, placebo-controlled Phase II clinical trial (RCT) to evaluate the efficacy of topical 10% Polyvinylpyrrolidone-Iodine (PVPI) to prevent new cavitated caries lesions when applied to the teeth of children with Severe Early Childhood Caries (S-ECC). At baseline, following oral rehabilitation in the operating room …

Accepts healthy volunteers
  • 0 views
  • 22 Sep, 2022
  • 1 location
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD) (Baby-COMET)  

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …

replacement therapy
deficiency
  • 6 views
  • 09 Sep, 2022
  • 9 locations
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD) (Baby-COMET)

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …

deficiency
replacement therapy
  • 0 views
  • 21 Sep, 2022
  • 9 locations
A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence

atrophy
geographic atrophy
pupillary dilation
vaccination
fundus autofluorescence imaging
  • 1382 views
  • 21 Sep, 2022
  • 84 locations
  • 73 views
  • 10 Aug, 2022
  • 63 locations
A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up

atrophy
geographic atrophy
  • 1 views
  • 19 Sep, 2022
  • 45 locations