Pompe-betegség: vizsgálatok itt: Egyesült Államok

In Egyesült Államok 13 Also abroad 30

13 trials · From ClinicalTrials.gov, checked 2026. november 11.. Ask the study team whether a trial fits you.

No phaseTaking people
Pompe Disease Registry Protocol
Where
Ann Arbor, Aurora, Baltimore, Birmingham, Boston, Camden, Charlotte, Charlottesville, Chicago, Cincinnati, Cleveland, Columbia, Columbus, Coral Gables, Dallas, Decatur, Detroit, Dublin, Durham, Fairfax, Fort Worth, Gainesville, Grand Rapids, Greenville, Hackensack, Helena, Hershey, Huntington, Indianapolis, Iowa City, Jacksonville, Kansas City, Las Vegas, Little Rock, Loma Linda, Los Angeles, Louisville, Milwaukee, Minneapolis, Morristown, Nashville, New Brunswick, New Haven, New Hyde Park, New Orleans, New York, Norfolk, Oakland, Omaha, Orange, Paterson, Philadelphia, Phoenix, Pittsburgh, Portland, Providence, Rochester, Sacramento, Salt Lake City, San Bernardino, San Diego, San Francisco, Seattle, South Burlington, Springfield, Stanford, St Louis, Syracuse, Tacoma, Tampa, Tucson, Valhalla, Washington D.C.
People
2000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00231400
1/2. fázisTaking people
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)
Where
Dallas, Durham, Irvine, New York, Palo Alto, Philadelphia, Phoenix, Pittsburgh, Richmond
Életkor
18 év to 65 év
People
12
Run by
AskBio Inc
See details and how to joinNCT07282847
1. fázisTaking people
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Where
Durham, Fairfax, Irvine, St Louis
Életkor
18 év to 75 év
People
32 worldwide
Run by
Denali Therapeutics Inc.
See details and how to joinNCT07354724 · EU 2025-524082-25-00
2. fázisTaking people
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Where
Atlanta, Cincinnati, Durham, Fairfax, Gainesville, Irvine, Philadelphia, Pittsburgh, St Louis
Életkor
18 év and over
People
45 worldwide
Run by
Shionogi
See details and how to joinNCT07123155
No phaseTaking people
Pompe Pregnancy Sub-Registry
Where
Atlanta, Dublin, Durham, Fairfax, Grand Rapids, Greenville, Indianapolis, Iowa City, Jacksonville, New York, Phoenix, Portland, St Louis
People
20 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00567073
No phaseTaking people
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Where
Durham
People
400
Run by
Duke University
See details and how to joinNCT01665326
No phaseTaking people
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Where
San Francisco
Életkor
Up to 64 év
People
250
Run by
University of California, San Francisco
See details and how to joinNCT05619900
1. fázisTaking people
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Where
San Francisco
Életkor
18 év to 50 év
People
10
Run by
University of California, San Francisco
See details and how to joinNCT04532047
No phaseTaking people
A Global Prospective Observational Registry of Patients With Pompe Disease
Where
Atlanta, Cincinnati, Durham, Fairfax, Indianapolis, Irvine, Jacksonville, Little Rock, New York, Philadelphia, Pittsburgh, Salt Lake City, St Louis
People
500 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT06121011
No phaseTaking people
Cognitive and Neurological Pathologies in Pompe Disease
Where
Durham
Életkor
0 év to 18 év
People
45
Run by
Duke University
See details and how to joinNCT04639336
3. fázisTaking people
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Where
Atlanta, Cincinnati, Durham, Gainesville, Pittsburgh, Salt Lake City
Életkor
Up to 17 év
People
36 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT04808505 · EU 2022-501095-25-01
No phaseTaking people
Avalglucosidase Alfa Pregnancy Study
Where
Bridgewater
People
100
Run by
Sanofi
See details and how to joinNCT05734521
No phaseTaking people
Long-Term Development of Muscular Dystrophy Outcome Assessments
Where
Richmond
Életkor
6 év to 50 év
People
1000
Run by
Virginia Commonwealth University
See details and how to joinNCT05989620