Also on Trial Friend:Pompe Disease pageMatch Me
All 20 Pompe Disease trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Pompe Disease Registry ProtocolMost sites in New York, California and OhioRecruitingGenzyme, a Sanofi Company72US sites
34 states - A Global Prospective Observational Registry of Patients With Pompe DiseaseMost sites in Pennsylvania, Ohio and ArkansasRecruitingAmicus Therapeutics15US sites
12 states - Pompe Pregnancy Sub-RegistryMost sites in New York, Arizona and FloridaRecruitingGenzyme, a Sanofi Company14US sites
12 states - A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)Most sites in California, Pennsylvania and ArizonaRecruitingPhase 1/2Ages 18-65AskBio9US sites
7 states - Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)Most sites in Pennsylvania, California and FloridaRecruitingPhase 2Ages 18+Shionogi9US sites
8 states - A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Most sites in Florida, Georgia and North CarolinaRecruitingPhase 3Up to age 17Amicus Therapeutics6US sites
6 states - A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseMost sites in California, Missouri and North CarolinaRecruitingPhase 1Ages 18-75Denali Therapeutics4US sites
4 states - Avalglucosidase Alfa Pregnancy StudyOnly in New JerseyRecruitingSanofi1US site
- Cognitive and Neurological Pathologies in Pompe DiseaseOnly in North CarolinaRecruitingAges 0-18Duke University1US site
- Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseOnly in North CarolinaRecruitingDuke University1US site
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Only in CaliforniaRecruitingPhase 1Ages 18-50University of California, San Francisco1US site
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesOnly in CaliforniaRecruitingUp to age 64University of California, San Francisco1US site
- A Long-term Study for the Outcome of Pompe DiseaseRecruitingNational Taiwan University HospitalNo US site yet
- China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDRecruitingPhase 4Up to age 17Genzyme, a Sanofi CompanyNo US site yet
- Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugRecruitingPhase 1/2Ages 6+GeneCradleNo US site yet
- Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic PathologyRecruitingAges 18+Centre Hospitalier Universitaire de LiegeNo US site yet
- Natural History of Pompe DiseaseRecruitingAges 18-80Assistance Publique - Hôpitaux de ParisNo US site yet
- Phrenic Nerve and Diaphragm Electrophysiology in Pompe DiseaseRecruitingAges 18+IRCCS National Neurological Institute "C. Mondino" FoundationNo US site yet
- Pompe Disease RWE Long-term Monitoring ProgramRecruitingNational Taiwan University HospitalNo US site yet
- Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle DiseasesRecruitingAssistance Publique Hopitaux De MarseilleNo US site yet
New trials, status changes and new sites, straight from ClinicalTrials.gov. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Who runs the Pompe Disease trials in the US, and what stage they are in
Of the 12 Pompe Disease trials with a US site, 1 is in Phase 3, the last stage before a company can ask the FDA for approval. Earlier Phase 1 or 2 tests of safety and dosing make up 4 of them. The remaining 7 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 8 of them, and universities, hospitals, foundations and government groups run 4. The sponsors with the most are Amicus Therapeutics (2), Duke University (2) and Genzyme, a Sanofi Company (2). The most recently listed one first appeared on ClinicalTrials.gov on January 21, 2026.
Pompe Disease trials in the US, state by state
Of the 12 Pompe Disease trials with a US site, 8 accept children under 18. Their open sites are in 33 states and Washington, DC, most often North Carolina, Florida and Georgia. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
Show the other 24 states
US hospitals running the most Pompe Disease trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Pompe Disease.
- Duke University Medical CenterDurham, North Carolina4 trials
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio2 trials
- Duke University Medical Center Genetics Dept- Site Number : 840037Durham, North Carolina2 trials
- O & O Alpan- Site Number : 840025Fairfax, Virginia2 trials
- Washington University School of MedicineSt Louis, Missouri2 trials
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011Chicago, Illinois1 trial
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013Chicago, Illinois1 trial
- Arkansas Children's Hospital- Site Number : 840109Little Rock, Arkansas1 trial
- Atlantic Health System- Site Number : 840099Morristown, New Jersey1 trial
- Atrium Health Carolinas Medical Center Hospital- Site Number : 840065Charlotte, North Carolina1 trial
Help getting to a Pompe Disease trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Pompe Disease trial near you
Are there Pompe Disease clinical trials near me?
As of November 13, 2026, 20 Pompe Disease clinical trials are recruiting now and 6 are not yet recruiting. In the US, 12 of them have at least 1 open site, across 33 states and Washington, DC. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Pompe Disease clinical trial sites?
Recruiting Pompe Disease trials list open sites in 33 states and Washington, DC. North Carolina, California and Virginia have the most, and the table on this page lists every state.
Are there Pompe Disease trials for children near me?
Of the 12 Pompe Disease trials with a US site, 8 accept children under 18. Their open sites are in 33 states and Washington, DC, most often North Carolina, Florida and Georgia. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Pompe Disease clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Pompe Disease trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Pompe Disease trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Pompe Disease: Lumizyme (alglucosidase alfa), Nexviazyme (avalglucosidase alfa), Pombiliti + Opfolda (cipaglucosidase alfa + miglustat).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.