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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.


Found (37) 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 purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) receiving standard-of-care enzyme replacement therapy (alglucosidase alfa; ERT).

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 ...


Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to ...

Phase N/A

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Subjects with Pompe disease who are scheduled to receive a NeuRx Diaphragm pacer (DPS) will be invited to participate in this study. The following tests will be performed during participation in the study: Forced expiratory tests Maximal Inspiratory Pressure (MIP) Resting Breathing Pattern Phrenic Nerve Function Evaluation with EMG Severe ...

Phase N/A

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with Late-Onset Pompe Disease (LOPD) who have tongue weakness. The results of this study will help design future research studies about LMT in LOPD.

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

The Effects of Regular Eye-training With a Mobile Device on Adult Patients With AMD

In the presented study a training-therapy in patients with dry AMD war evaluated. The main focus was on remote visibility and thus better handling of daily activities. Therapy with high-contrast administration is used for amblyopia in childhood. Furthermore, simulation therapy is used in hemianopsia in the adult age, in which ...

Phase N/A

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness

A breach of respiratory function may be one of the elements more or less early or predominant clinical picture of neuromuscular diseases. It is considered that the obstructive syndromes represent 64% and restrictive or mixed syndromes 36% of chronic respiratory insufficiency, approximately 7% due to a neuromuscular disease. The frequency ...

Phase N/A

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 N/A