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 151 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)

  • 584 views
  • 23 Nov, 2020
  • 1 location
Featured trial
A Phase 3 Multicenter, Randomized, Double Masked, Sham- Controlled Clinical Trial to Assess the Safety and Efficacy of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

The objectives of this study are to evaluate the safety and efficacy of Zimura intravitreal administration in patients with geographic atrophy secondary to age-related macular degeneration (AMD

  • 6 views
  • 24 Jun, 2021
  • 1 location
None
Double-Masked Study of Photrex (Rostaporfin) Photodynamic Therapy in the Treatment of Age-Related Macular Degeneration

neovascularization (CNV) associated with age-related macular degeneration (AMD).

fluorescein angiography
neovascularization
angiography
photodynamic therapy
age-related macular degeneration
  • 56 views
  • 07 Nov, 2020
  • 1 location
None
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
None
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-nave pediatric subjects with classic IOPD.

  • 0 views
  • 03 Nov, 2021
  • 1 location
None
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease

replacement therapy
gene therapy
  • 29 views
  • 25 Jul, 2021
  • 21 locations
None
Intravitreal Bevacizumab Combined With PDT Versus Bevacizumab to Treat Exudative AMD

The purpose of this study is to determine the association of bevacizumab and PDT is safety and effective in the treatment of exudative AMD

bevacizumab
corrected visual acuity
neovascularization
  • 10 views
  • 07 Nov, 2020
  • 1 location
None
Treatment of Patients With Neovascular AMD Using Indocyanine Green-Mediated Photothrombosis (i-MP).

To establish the safety and efficacy of indocyanine green-mediated photothrombosis (i-MP) for the treatment of patients with neovascular age-related macular degeneration.

fluorescein angiography
indocyanine green
wet age-related macular degeneration
drusen
neovascularization
  • 22 views
  • 07 Nov, 2020
  • 1 location
None
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
  • 18 Jun, 2021
  • 4 locations
None
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

spectral domain optical coherence tomography
drusen
  • 0 views
  • 22 Oct, 2021
  • 1 location