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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 (22) clinical trials

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

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

Pompe Lactation Sub-Registry

The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa.

Phase

Pompe Pregnancy Sub-Registry

Study Design Time Perspective: Retrospective and Prospective

Phase

Immune Tolerance Induction Study

An exploratory, open-labeled study of patients with Pompe disease, who have previously received Myozyme (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme. Eligible patients who are currently receiving Myozyme therapy will be enrolled into the study, ...

Phase

A Long Term Follow up Study in Late-onset Pompe Disease

This study is to observe the progression of disease in late-onset Pompe disease

Phase N/A

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy

Phase N/A

Pompe Disease Registry

The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives ...

Phase N/A

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

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