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Pompe Disease Clinical Trials

A listing of Pompe Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (39) clinical trials

The Comet Study may be appropriate for those who meet the following criteria: • Three years of age or older • Able to walk 40 meters (approximately 130 feet) without stopping and without an assistive device • Has not had previous treatment with immune tolerance induction therapy, alglucosidase alfa, or ...

Phase

The Rare Disease Registry Program (including but not limited to, Gaucher, Fabry, MPS I, and Pompe diseases) is a multi-center, international, longitudinal, observational program that tracks the natural history and outcomes of patients with these rare diseases.

Phase N/A

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

The following tests will be completed over a two-day period: Respiratory pressure tests, breathing test, magnetic resonance imaging and magnetic resonance spectroscopy (MRI and MRS).

Phase N/A

Biomarker for Glycogen Storage Diseases

Glycogen storage diseases (GSDs) are a group of inherited genetic disorders that cause glycogen to be improperly stored in the body. People with glycogen storage diseases have a buildup of abnormal amounts or types of glycogen in their tissues. The main types of glycogen storage diseases are categorized by number ...

Phase N/A

Identification of Tongue Involvement in Late-Onset Pompe Disease

This purpose of this study is to determine if tongue strength and tongue ultrasound measurements differentiates patients with untreated late-onset Pompe Disease (LOPD) from patients with acquires/hereditary myopathies or neuropathies. It is hypothesized that abnormalities in tongue function and structure in patients with LOPD may be useful in discriminating this ...

Phase N/A

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

The duration of the study per patient will be up to 3 years that will consist of a 14- day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period, a 96-week open-label treatment period, and a 4-week post-treatment observation period.

Phase

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

The incidence of type II glycogen-storage disease (Pompe disease) varies depending on ethnicity and geographic region. As of 2010, nine studies have been published documenting the incidence of Pompe disease. It is most common within the African American population, with an incidence of 1 in 14,000. In the U.S. more ...

Phase N/A

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease

Patients will be enrolled in a consecutive manner and randomized to either treatment with VAL-1221 or positive control (rhGAA) in cohorts of 4 patients each: Part 1 Study Treatment: Cohort 1: VAL-1221 3 mg/kg IV (n=3) every other week or control (n=1) Cohort 2: VAL-1221 10 mg/kg IV (n=3) every ...

Phase

A Pilot Study of Zavesca in Patients With Pompe Disease and Infusion Associated Reaction

This Study is designed to assess the effects of Zavesca as immunomodulatory therapy on anti-rhGAA immune responses in patients with Pompe disease, as well as their health and disease progression. Subjects will either receive Zavesca at 100 mg or 300 mg dosing levels during study participation (n=3 @ 100 mg ...

Phase

A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI

Study aim: The principal objective of the study is to find biomarkers that quantify the natural progression of the disease and to know if they are useful to determine the improvement or lack of impairment of the disease in response to Enzyme Replacement Therapy (ERT). Study design: A single center ...

Phase N/A