Pompe disease: open trials in United States
14 open trials have a site in United States. Registry data; only the trial team decides who can take part.
NCT00231400
Pompe Disease Registry Protocol
Research · Genzyme, a Sanofi Company
University Of Michigan Pediatrics- Site Number : 840107, Ann Arbor · Children's Hospital Colorado - Aurora- Site Number : 840069, Aurora · University Of Colorado At Denver Genetics- Site Number : 840068, Aurora · Baltimore · University of Alabama at Birmingham- Site Number : 840106, Birmingham · Boston Children's Hospital- Site Number : 840092, Boston · Massachusetts General Hospital-Genetics- Site Number : 840062, Boston · Cooper Health Center- Site Number : 840098, Camden · Atrium Health Carolinas Medical Center Hospital- Site Number : 840065, Charlotte · University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078, Charlottesville · Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011, Chicago · Rush University Medical Center Genetics- Site Number : 840079, Chicago · Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013, Chicago · Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840041, Cincinnati · Cleveland Clinic - Cleveland- Site Number : 840048, Cleveland · University Hospitals Cleveland Medical Center- Site Number : 840119, Cleveland · University Of Missouri Health System Department Of Genetics- Site Number : 840031, Columbia · Nationwide Children's Hospital- Site Number : 840091, Columbus · University of Miami- Site Number : 840006, Coral Gables · Baylor Institute of Metabolic Disease- Site Number : 840058, Dallas · Emory University School Of Medicine- Human Genetics- Site Number : 840060, Decatur · Children's Hospital of Michigan- Site Number : 840066, Detroit · Lsd Data Registry Site Llc- Site Number : 840094, Dublin · Duke University Medical Center Genetics Dept- Site Number : 840037, Durham · O & O Alpan- Site Number : 840025, Fairfax · Cook Children's Medical Center- Site Number : 840024, Fort Worth · University of Florida College of Medicine- Site Number : 840012, Gainesville · Infusion Associates - Grand Rapids- Site Number : 840050, Grand Rapids · Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019, Grand Rapids · Greenwood Genetic Center - Greenville- Site Number : 840055, Greenville · Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 840101, Hackensack · Shodair Children'S Hospital- Site Number : 840090, Helena · Penn State Health Milton South Hershey Medical Center- Site Number : 840061, Hershey · NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040, Huntington · Indianapolis University School Of Medicine- Site Number : 840027, Indianapolis · University of Iowa- Site Number : 840032, Iowa City · The Atwal Clinic- Site Number : 840112, Jacksonville · University Of Florida Pediatrics Genetics- Site Number : 840121, Jacksonville · University of Kansas Medical Center- Site Number : 840020, Kansas City · Children's Mercy Adelle Hall Campus- Site Number : 840063, Kansas City · Children's Specialty Center - Las Vegas- Site Number : 840008, Las Vegas · University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock · Arkansas Children's Hospital- Site Number : 840109, Little Rock · Loma Linda University- Genetics- Site Number : 840070, Loma Linda · Southern California Permanente Medical Group- Site Number : 840108, Los Angeles · Usc Health Sciences Center Dept Of Genetics- Site Number : 840082, Los Angeles · University of Louisville - Health Sciences Center- Site Number : 840030, Louisville · Milwaukee · M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076, Minneapolis · Children's Hospitals and Clinics of Minnesota- Site Number : 840046, Minneapolis · M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840035, Minneapolis · Children's Hospitals and Clinics of Minnesota- Site Number : 840114, Minneapolis · Atlantic Health System- Site Number : 840099, Morristown · Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049, Nashville · Saint Peter's University Hospital - New Brunswick- Site Number : 840016, New Brunswick · Yale University School of Medicine- Site Number : 840047, New Haven · Northwell Health- Site Number : 840102, New Hyde Park · Tulane University School of Medicine- Site Number : 840001, New Orleans · Ochsner Medical Center - Jefferson Highway- Site Number : 840120, New Orleans · Icahn School of Medicine at Mount Sinai- Site Number : 840005, New York · Columbia University Irving Medical Center- Site Number : 840125, New York · Metropolitan Hospital Center- Site Number : 840110, New York · Children's Hospital of The King's Daughters- Site Number : 840072, Norfolk · UCSF Benioff Children's Hospital - Oakland- Site Number : 840029, Oakland · University of Nebraska Medical Center- Site Number : 840084, Omaha · Children's Hospital of Orange County- Site Number : 840074, Orange · University of California Irvine Medical Center- Site Number : 840036, Orange · St. Joseph's Regional Medical Center - Paterson- Site Number : 840057, Paterson · Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089, Philadelphia · The Children's Hospital of Philadelphia- Site Number : 840034, Philadelphia · Phoenix Children's Hospital- Site Number : 840003, Phoenix · Barrow Neurological Institute- Site Number : 840087, Phoenix · UPMC Children's Hospital of Pittsburgh- Site Number : 840014, Pittsburgh · UPMC Children's Hospital of Pittsburgh- Site Number : 840023, Pittsburgh · Maine Medical Partners Pediatric Specialty Care- Site Number : 840064, Portland · Oregon Health & Science University (OHSU)- Site Number : 840080, Portland · Oregon Health & Science University (OHSU)- Site Number : 840038, Portland · Oregon Health & Science University (OHSU)- Site Number : 840095, Portland · Rhode Island Hospital Genetics- Site Number : 840053, Providence · University of Rochester Medical Center- Site Number : 840105, Rochester · Kaiser Permanente Sacramento Medical Center- Site Number : 840042, Sacramento · Uc Davis Mind Institute- Site Number : 840010, Sacramento · University of Utah- Site Number : 840043, Salt Lake City · Loma Linda University Children'S Hospital- Site Number : 840117, San Bernardino · University of California at San Diego- Site Number : 840007, San Diego · University of California San Francisco - Parnassus Campus- Site Number : 840051, San Francisco · Seattle Children's Hospital- Site Number : 840028, Seattle · University of Washington Medical Center- Site Number : 840059, Seattle · The University of Vermont Medical Center- Site Number : 840129, South Burlington · Baystate Medical Center- Site Number : 840002, Springfield · Stanford University Medical Center- Site Number : 840021, Stanford · Washington University- Site Number : 840100, St Louis · SUNY Upstate Medical University - Site Number : 840052, Syracuse · Mary Bridge Children's Hospital & Health Center- Site Number : 840115, Tacoma · University Of Florida-Genetics- Site Number : 840096, Tampa · University of Arizona- Site Number : 840015, Tucson · New York Medical College- Site Number : 840039, Valhalla · Children's National Medical Center - Washington- Site Number : 840067, Washington D.C.
Registry updated 2026-10-28
NCT07282847
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)
Trial Treatment · Phase 1/2 · AskBio Inc
University of Texas Southwest Medical Center, Dallas · Duke University, Durham · University of California, Irvine (UCI), Irvine · NYU Langone, New York · Stanford Neuroscience Health Center, Palo Alto · Hospital of the University of Pennsylvania, Philadelphia · Barrow Neurological Institute, Phoenix · University of Pittsburgh Medical Center (UPMC), Pittsburgh · Virginia Commonwealth University (Vcu), Richmond
Registry updated 2026-10-13
NCT07354724
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Trial Treatment · Phase 1 · Denali Therapeutics Inc.
Duke University School of Medicine - Early Phase Research Unit, Durham · The Lysosomal & Rare Disorders Research & Treatment Center, Fairfax · University of California, Irvine, Irvine · Washington University School of Medicine, St Louis
Registry updated 2026-08-17
NCT07123155
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Trial Treatment · Phase 2 · Shionogi
Emory University Hospital, Atlanta · Cincinnati Children's Hospital Medical Center, Cincinnati · Duke University Medical Center, Durham · Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), Fairfax · University of Florida (UF) - Gainesville, Gainesville · University of California, Irvine Medical Center, Irvine · University of Pennsylvania, Philadelphia · University of Pittsburgh School of Medicine, Pittsburgh · Washington University in St. Louis, St Louis
Registry updated 2026-06-23
NCT00567073
Pompe Pregnancy Sub-Registry
Research · Genzyme, a Sanofi Company
Emory University School Of Medicine- Site Number : 840060, Atlanta · Lsd Data Registry Site Llc- Site Number : 840094, Dublin · Duke University Medical Center Genetics Dept- Site Number : 840037, Durham · O & O Alpan- Site Number : 840025, Fairfax · Spectrum for Health- Site Number : 840019, Grand Rapids · Greenwood Genetic Center - Greenville- Site Number : 151184, Greenville · Indianapolis University School Of Medicine- Site Number : 840027, Indianapolis · University of Iowa- Site Number : 150294, Iowa City · University of Florida Pediatrics Genetics- Site Number : 157138, Jacksonville · Mt. Sinai School of Medicine- Site Number : 840005, New York · New York University School Of Medicine- Site Number : 840040, New York · Columbia University Irving Medical Center- Site Number : 157199, New York · Phoenix · Oregon Health and Science University- Site Number : 840095, Portland · Washington University- Site Number : 150612, St Louis
Registry updated 2026-04-16
NCT01665326
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Research · Duke University
Duke University Medical Center, Durham
Registry updated 2026-04-08
NCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Research · University of California, San Francisco
University of California San Francisco, San Francisco
Registry updated 2026-04-08
NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Trial Treatment · Phase 1 · University of California, San Francisco
University of California, San Francisco
Registry updated 2026-03-17
NCT07478172
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Trial Treatment · University of Missouri-Columbia
NextGen Precision Health Building, Clinical and Translational Science Unit, Columbia
Registry updated 2026-03-17
NCT06121011
A Global Prospective Observational Registry of Patients With Pompe Disease
Research · Amicus Therapeutics
Emory University, Atlanta · University of Cincinnati Medical Center, Cincinnati · Cincinnati Children's Hospital, Cincinnati · Duke University Medical Center, Durham · Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax · Indiana University, IU Health Physicians Neurology, Indianapolis · University of California, Irvine, Irvine · Wolfson Children's Hospital, Jacksonville · University of Arkansas Medical Science, Little Rock · NYU Neurogenetics, NYU Langone Medical Center, New York · University of Pennsylvania - Perelman Center for Advanced Medicine, Philadelphia · UPMC Children's Hospital of Pittsburgh, Pittsburgh · University of Pittsburgh, Pittsburgh · University of Utah, Salt Lake City · Washington University School of Medicine, St Louis
Registry updated 2026-03-10
NCT04639336
Cognitive and Neurological Pathologies in Pompe Disease
Research · Duke University
Duke University Medical Center, Durham
Registry updated 2026-03-04
NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Trial Treatment · Phase 3 · Amicus Therapeutics
The Emory Clinic, Atlanta · Cincinnati Children's Hospital Medical Center, Cincinnati · Duke University Early Phase Research Unit, Durham · University of Florida Clinical Research Center, Gainesville · UPMC Hospital of Pittsburgh, Pittsburgh · University of Utah, Clinical and Translational Sciences Institute, Salt Lake City
Registry updated 2026-01-22
NCT05734521
Registry updated 2026-01-16
NCT05989620
Long-Term Development of Muscular Dystrophy Outcome Assessments
Research · Virginia Commonwealth University
Virginia Commonwealth University, Richmond
Registry updated 2025-11-18
All Pompe disease trials and the map
This page is not medical advice. Talk to your doctor before contacting a trial.