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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 (34) 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

This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.  


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

Resveratrol for Exudative Age-Related Macular Degeneration

Interventional, prospective, randomized, comparative monocentric study aiming to evaluate the safety and efficacy of Resveratrol to reduce the progression of exudative Age-Related Macular Degeneration. Comparison of incidence of choroidal neaovascularization between each study group will be performed.

Phase N/A

Investigating Lysosomal Storage Diseases in Minority Groups

Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of ...

Phase N/A

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Patients will be compared to a group of matched healthy control subjects (matched for age, gender, physical activity level and BMI) for comparison of the primary and secondary outcome measures. Methods Each subject will go through 3 test days. Test day 1: International Physical Activity Questionnaire (IPAQ). The results will ...

Phase N/A

A Long-term Study for the Outcome of Pompe Disease

Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.

Phase N/A