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.

Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 129 clinical trials
Featured trial
Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)

Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)

  • 592 views
  • 23 Nov, 2020
  • 1 location
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

  • 0 views
  • 31 Oct, 2021
  • 1 location
A Study to Evaluate the Safety and Efficacy of RQC for AMD

To evaluate the safety and efficacy of resveratrol, quercetin, and curcumin in combination (RQC) over 2 years in patients with age-related macular degeneration (AMD).

geographic atrophy
drusen
atrophy
  • 0 views
  • 06 Oct, 2021
Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with classic IOPD.

  • 14 views
  • 24 Mar, 2022
  • 1 location
Zoledronic Acid as Adjuvant Therapy in Neovascular Age-related Macular Degeneration (Z-AMD) (Z-AMD)

A pilot study of zoledronic acid as adjuvant therapy to standard anti-vascular endothelial growth factor (anti-VEGF) treatment for neovascular age-related macular degeneration (AMD).

  • 0 views
  • 24 Mar, 2022
  • 2 locations
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

drusen
spectral domain optical coherence tomography
  • 0 views
  • 22 Oct, 2021
  • 1 location
Phase I Study to Compare CMAB818 Injection and Lucentis® in Patients With Wet AMD

This is a randomized, double-blind, two-group parallel, positive-controlled clinical Phase I trial comparing the safety, pharmacokinetics, pharmacodynamics and efficacy of CMAB818 and Lucentis® in patients with wet age-related macular degeneration.

  • 0 views
  • 05 Jul, 2022
  • 1 location
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 Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular degeneration (AMD) eyes to new incomplete retinal pigment

atrophy
retinal pigment
optical coherence tomography
  • 2 views
  • 15 Jun, 2022
  • 6 locations
Safety and Tolerability of RPESC-derived 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.

  • 0 views
  • 27 Apr, 2022
  • 1 location