Clinical Trials for Pompe Disease (Late-onset)

Find clinical trials for pompe disease (late-onset). Discover research opportunities and treatment options in your area.

9 Active Trials Found

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A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT ID: NCT06150820

This clinical trial is studying people with late-onset Pompe disease, a genetic condition that causes muscle weakness over time. The researchers want to learn about the levels of antibodies against...

Recruiting

Phase: NA Type: interventional Locations: Kansas City, Kansas, United States, Ann Arbor, Michigan, United States +53 more

Pompe Pregnancy Sub-Registry

NCT ID: NCT00567073

**Summary of the Pompe Pregnancy Sub-Registry** The Pompe Pregnancy Sub-Registry is a study aimed at understanding how pregnancy affects women with Pompe disease, which is a rare genetic condition...

Recruiting

Type: observational Locations: Zagreb, , Croatia, Zagreb, , Croatia +75 more

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT ID: NCT03911505

The ZIP Study-OL is a clinical trial that aims to evaluate the safety and effectiveness of a new treatment called Cipaglucosidase Alfa/Miglustat for children and teenagers with Pompe disease, speci...

Recruiting

Phase: PHASE3 Type: interventional Locations: Rochester, Minnesota, United States, Durham, North Carolina, United States +21 more

Treatment Frequency Reduction in Pompe Disease

NCT ID: NCT06575829

This clinical trial is studying whether patients with late-onset Pompe disease can safely reduce their treatment frequency of alglucosidase alfa from every two weeks to every four weeks, without wo...

Not yet recruiting

Phase: PHASE4 Type: interventional

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

NCT ID: NCT06391736

This clinical trial is investigating a new gene therapy drug called GC301, which is designed to treat late-onset Pompe disease (LOPD). Pompe disease is a rare genetic condition that affects how the...

Recruiting

Phase: PHASE1 Type: interventional Locations: Beijing, , China

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

NCT ID: NCT04929002

This clinical trial is looking at how high levels of glycogen, a type of sugar stored in muscles, might be related to muscle damage in patients with certain Glycogen Storage Diseases, like McArdle ...

Recruiting

Type: observational Locations: Copenhagen, Copenhange, Denmark

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

NCT ID: NCT06178432

This clinical trial is investigating a new gene therapy treatment called CRG003 for adults with late-onset Pompe disease (LOPD). Pompe disease is a rare genetic condition that affects the muscles a...

Not yet recruiting

Phase: EARLY_PHASE1 Type: interventional Locations: Shanghai, , China

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

NCT ID: NCT05272969

The "Pompe & Pain" study is looking at how common and severe muscle pain is in adults with late-onset Pompe disease, a condition that affects muscle function. Researchers want to understand not jus...

Recruiting

Type: observational Locations: Munich, Bavaria, Germany

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

NCT ID: NCT05951790

This clinical trial is studying the effects of a special exercise program called Inspiratory Muscle Training (IMT) for adults with Late-onset Pompe Disease, a condition that affects muscle strength...

Recruiting

Phase: NA Type: interventional Locations: Milan, , Italy

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