A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a....
A Study to Evaluate the Safety, Efficacy, PK, 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, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric sub...
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygou...
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experience...
Top Cities for Pompe Disease Clinical Trials
Pompe Disease clinical trials are recruiting across 24 cities. Here are the cities with the most active studies:
About Pompe Disease
Pompe disease is a rare genetic disorder caused by accumulation of glycogen in cells due to deficiency of the enzyme acid alpha-glucosidase. It can affect the heart, skeletal muscles, and respiratory system. Treatment includes enzyme replacement therapy.
Clinical trials are advancing new treatments for pompe disease. Currently, 4 studies are recruiting a combined 139 participants across the United States. Research is being conducted by 3 organizations including ModernaTX, Inc., Amicus Therapeutics, Beam Therapeutics Inc..
2026 Pompe Disease Research Landscape
As of March 2026, the pompe disease clinical trial landscape includes 4 actively recruiting studies across 24 cities in the United States. These studies are collectively seeking 139 participants, with an average enrollment target of 35 per study.
Research is being led by 3 different organizations, including ModernaTX, Inc., Amicus Therapeutics, Beam Therapeutics Inc..
Geographically, pompe disease trials are most concentrated in Durham, North Carolina (3 trials); Farmington, Connecticut (2 trials); Cincinnati, Ohio (2 trials); Houston, Texas (2 trials); Salt Lake City, Utah (2 trials) and 7 other cities.
Featured Pompe Disease Studies
Highlighted recruiting studies for pompe disease, selected by enrollment size and research scope.
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.
A Study to Evaluate the Safety, Efficacy, PK, 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, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.
Frequently Asked Questions About Pompe Disease Clinical Trials
Are there pompe disease clinical trials near me?
Yes, there are 4 pompe disease clinical trials currently recruiting across 24+ cities in the United States, including Durham, North Carolina; Farmington, Connecticut; Cincinnati, Ohio. Browse the studies above to find one at a location convenient for you.
How do I join a pompe disease clinical trial?
To join a pompe disease clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are pompe disease clinical trials free?
Yes, participation in pompe disease clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of pompe disease treatments are being studied?
Current pompe disease clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 3 research organizations.
Is it safe to participate in pompe disease clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 1, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 1, 2026 · Data from ClinicalTrials.gov