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 37 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
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.

  • 0 views
  • 24 Mar, 2022
  • 1 location
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
  • 81 views
  • 17 Mar, 2022
  • 9 locations
  • 17 views
  • 29 Oct, 2021
  • 14 locations
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (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
  • 0 views
  • 15 May, 2022
  • 7 locations
Quality-Assured Follow-up of Quiescent Neovascular Age -Related maculaR dEgeneration by Non-medical Practitioners

This is a prospective, randomised, multi-site clinical trial testing the non-inferiority of community optometry follow-up of participants with QnAMD over 12 months

macular degeneration
optical coherence tomography
  • 57 views
  • 27 Sep, 2021
  • 1 location
A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry

the epidemiology of MPS1 and Pompe disease using dried blood samples in the first cohort of neonates tested in France (Normandie region).

  • 0 views
  • 30 Oct, 2021
  • 2 locations
AAV2/8-LSPhGAA in Late-Onset Pompe Disease

Open-label, ascending dose trial of AAV2/8-LSPhGAA administered intravenously.

administered intravenously
hysterectomy
gaa gene
vasectomy
tubal ligation
  • 0 views
  • 05 Mar, 2021
  • 1 location
Gene Transfer Study in Patients With Late Onset Pompe Disease (FORTIS)

Late Onset Pompe Disease (LOPD).

replacement therapy
alglucosidase alfa
gaa gene
  • 0 views
  • 01 May, 2022
  • 4 locations
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

)] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective

myozyme
gaa gene
deficiency
alglucosidase alfa
beta-human chorionic gonadotropin
  • 0 views
  • 06 May, 2022
  • 1 location