Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Pompe Disease Clinical Trials
Pompe Disease clinical trials are recruiting across 35 cities. Here are the cities with the most active studies:
Pompe Disease Trial Phases
10 research organizations are sponsoring pompe disease trials.
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, 17 studies are recruiting a combined 4,186 participants across the United States. Research is being conducted by 10 organizations including Genzyme, a Sanofi Company, Amicus Therapeutics, Duke University and 7 others.
2026 Pompe Disease Research Landscape
As of July 2026, the pompe disease clinical trial landscape includes 17 actively recruiting studies across 35 cities in the United States. These studies are collectively seeking 4,186 participants, with an average enrollment target of 246 per study.
The research spans multiple phases of development: 3 studies are in PHASE2, 3 studies are in PHASE1, 1 study is in PHASE3. The presence of Phase 3 trials indicates that some pompe disease treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 10 different organizations, including Genzyme, a Sanofi Company, Amicus Therapeutics, Duke University, Ultragenyx Pharmaceutical Inc, Sanofi, and 5 others.
Geographically, pompe disease trials are most concentrated in Durham, North Carolina (12 trials); Irvine, California (5 trials); Atlanta, Georgia (4 trials); Philadelphia, Pennsylvania (4 trials); Phoenix, Arizona (3 trials) and 7 other cities.
Featured Pompe Disease Studies
Highlighted recruiting studies for pompe disease, selected by enrollment size and research scope.
Pompe Disease Registry Protocol
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and ...
A Global Prospective Observational Registry of Patients With Pompe Disease
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe dise...
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies ...
Frequently Asked Questions About Pompe Disease Clinical Trials
Are there pompe disease clinical trials near me?
Yes, there are 17 pompe disease clinical trials currently recruiting across 35+ cities in the United States, including Durham, North Carolina; Irvine, California; Atlanta, Georgia. 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 across PHASE2 (3 studies), PHASE1 (3 studies), PHASE3 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 10 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 July 17, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
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 July 17, 2026 · Data from ClinicalTrials.gov