Pompe Disease Clinical Trials Near Me
As of October 12, 2026, 26 Pompe Disease clinical trials are recruiting or about to open (21 recruiting now, 5 opening soon). 13 of them have at least 1 open site in the United States, across 32 states. Enter your ZIP code to see which are closest to you.
Also on Trial Friend:Pompe Disease pageMatch Me
All 21 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 ProtocolUniversity of Alabama at Birmingham- Site Number : 840106, Birmingham, Alabama; Barrow Neurological Institute- Site Number : 840087, Phoenix, Arizona; Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona and 71 more sitesRecruitingRun by Genzyme, a Sanofi Company
- A Global Prospective Observational Registry of Patients With Pompe DiseaseUniversity of Arkansas Medical Science, Little Rock, Arkansas; University of California Irvine, Irvine, California; Wolfson Children's Hospital, Jacksonville, Florida and 12 more sitesRecruitingRun by Amicus Therapeutics
- Pompe Pregnancy Sub-RegistryBarrow Neurol Group- Site Number : 840087, Phoenix, Arizona; University of Florida Pediatrics Genetics- Site Number : 157138, Jacksonville, Florida; Emory University School Of Medicine- Site Number : 840060, Atlanta, Georgia and 11 more sitesRecruitingRun by Genzyme, a Sanofi Company
- 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)Barrow Neurological Institute, Phoenix, Arizona; University of California, Irvine (UCI), Irvine, California; Stanford Neuroscience Health Center, Palo Alto, California and 6 more sitesRecruitingPhase 1/2Ages 18 to 65Run by AskBio Inc
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)University of California - Irvine Medical Center, Irvine, California; University of Florida (UF) - Gainesville, Gainesville, Florida; Emory University Hospital, Atlanta, Georgia and 6 more sitesRecruitingPhase 2Ages 18+Run by Shionogi
- A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18University of Florida Clinical Research Center, Gainesville, Florida; The Emory Clinic, Atlanta, Georgia; Duke University Early Phase Research Unit, Durham, North Carolina and 3 more sitesRecruitingPhase 3Up to age 17Run by Amicus Therapeutics
- A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseUniversity of California-Irvine, Irvine, California; Washington University School of Medicine, St Louis, Missouri; Duke University School of Medicine - Early Phase Research Unit, Durham, North Carolina and 1 more siteRecruitingPhase 1Ages 18 to 75Run by Denali Therapeutics Inc.
- Avalglucosidase Alfa Pregnancy Study1 open siteRecruitingRun by Sanofi
- Cognitive and Neurological Pathologies in Pompe DiseaseDuke University Medical Center, Durham, North CarolinaRecruitingAges 0 to 18Run by Duke University
- Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseDuke University Medical Center, Durham, North CarolinaRecruitingRun by Duke University
- Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseNextGen Precision Health Building, Clinical and Translational Science Unit, Columbia, MissouriRecruitingAges 18+Run by University of Missouri-Columbia
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)University of California, San Francisco, CaliforniaRecruitingPhase 1Ages 18 to 50Run by University of California, San Francisco
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesUniversity of California San Francisco, San Francisco, CaliforniaRecruitingUp to age 64Run by University of California, San Francisco
- A Long-term Study for the Outcome of Pompe DiseaseNo open US site listed yetRecruitingRun by National Taiwan University Hospital
- China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDNo open US site listed yetRecruitingPhase 4Up to age 17Run by Genzyme, a Sanofi Company
- Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugNo open US site listed yetRecruitingPhase 1/2Ages 6+Run by GeneCradle Inc
- Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic PathologyNo open US site listed yetRecruitingAges 18+Run by Centre Hospitalier Universitaire de Liege
- Natural History of Pompe DiseaseNo open US site listed yetRecruitingAges 18 to 80Run by Assistance Publique - Hôpitaux de Paris
- Phrenic Nerve and Diaphragm Electrophysiology in Pompe DiseaseNo open US site listed yetRecruitingAges 18+Run by IRCCS National Neurological Institute "C. Mondino" Foundation
- Pompe Disease RWE Long-term Monitoring ProgramNo open US site listed yetRecruitingRun by National Taiwan University Hospital
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Where Pompe Disease trials are recruiting in the US
The US cities with the most Pompe Disease trials recruiting are Durham, North Carolina (9), Fairfax, Virginia (5), Irvine, California (5), Pittsburgh, Pennsylvania (5) and St Louis, Missouri (5), counting trials with at least 1 open site in each city.
Hospitals 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, even if no trial fits you.
- Duke University Medical Center
- Duke University Medical Center Genetics Dept- Site Number : 840037
- Duke University
- and 2 more
- O & O Alpan- Site Number : 840025
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- and 1 more
- University of California - Irvine Medical Center
- University of California at Irvine- Site Number : 840036
- University of California Irvine
- and 2 more
- University of Pittsburgh
- University of Pittsburgh Medical Center (UPMC)
- University of Pittsburgh School of Medicine
- and 4 more
- Washington University School of Medicine
- Washington University in St. Louis
- Washington University- Site Number : 150612
- and 1 more
- Emory University
- Emory University Hospital
- Emory University School Of Medicine- Site Number : 840060
- and 1 more
- Cincinnati Children's Hospital Medical Center
- Cincinnati Children's Hospital
- University of Cincinnati Medical Center
- and 1 more
- Columbia University Irving Medical Center- Site Number : 157199
- Columbia University Irving Medical Center- Site Number : 840125
- Icahn School of Medicine at Mount Sinai- Site Number : 840005
- and 5 more
States with at least 1 open site: Alabama, Arizona, Arkansas, California, Connecticut, District of Columbia, Florida, Georgia, Illinois, Indiana, Iowa, Kansas, Maine, Massachusetts, Michigan, Minnesota, Missouri, Nebraska, Nevada, New Jersey, New York, North Carolina, Ohio, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Utah, Virginia, Washington, Wisconsin.
Questions about finding a Pompe Disease trial near you
Are there Pompe Disease clinical trials near me?
As of October 12, 2026, 26 Pompe Disease clinical trials are recruiting or about to open (21 recruiting now, 5 opening soon). 13 of them have at least 1 open site in the United States, across 32 states. 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?
As of October 12, 2026, recruiting Pompe Disease trials list open sites in 32 states. The US cities with the most Pompe Disease trials recruiting are Durham, North Carolina (9), Fairfax, Virginia (5), Irvine, California (5), Pittsburgh, Pennsylvania (5) and St Louis, Missouri (5), counting trials with at least 1 open site in each city.
What if there is no Pompe Disease trial near me?
Some trials pay for or reimburse travel and lodging, and a few send staff or use local labs so fewer visits happen at the study site, so ask the study contact before ruling a trial out. 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: Nexviazyme (avalglucosidase alfa), Lumizyme (alglucosidase alfa), Pombiliti + Opfolda (cipaglucosidase alfa + miglustat).
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of October 12, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out.