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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 (36) clinical trials

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.  

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

Cell Collection to Study Eye Diseases

This study will establish a repository of biospecimens to generate induced pluripotent stem (iPS) cells, which will be used to determine molecular mechanisms for potentially blinding eye diseases including but not limited to: Best Vitelliform Dystrophy (Best Disease); Late-Onset Retinal Degeneration (L-ORD); Age-Related Macular Degeneration (AMD); Leber congenital amaurosis (LCA); ...

Phase N/A

CNV in AMD Analyzed by OCT Angiography Under IntravitreaL Eylea (COCTAEyl)

The main objective of this study is to analyze a new noninvasive imaging examination, OCT angiography, in the evaluation of neovascular remodeling and early signs of recurrence of wet AMD undergoing treatment on OCTA and to correlate OCTA findings with SD-OCT findings.

Phase N/A

Retinal Imaging Using NOTAL-OCT V3.0

4.5.1 SCREENING VISIT: For patients eligible to participate in the study, the following procedure will be done in the screening visit: Patient will sign the IC (Informed Consent) form Test the eligible eye(s) of the patient will be tested The following demographic and clinical data will be collected for each ...

Phase N/A

ZIP Study - A Study to Evaluate the PK Safety Efficacy and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18

The study will consist of a 30-day screening period, a 12-month treatment period, and a 30-day safety follow-up period, for a total duration of approximately 14 months. Subjects who complete this study may have an opportunity to enroll in a separate long-term extension study. Pediatric subjects will be treated every ...

Phase

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

This is an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Phase

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