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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 (40) 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

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

A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

The objective of this study is to evaluate the baseline characteristics and degree of change over time in clinical outcome measures commonly used to evaluate patients with LOPD.

Phase N/A

Diet and Exercise in Pompe Disease

Pompe is a rare disease, which occurs in approximately 1 in 40,000 births. It is a progressive and often fatal neuromuscular disorder resulting from a mutation in the gene for acid alpha- glucosidase (GAA), an enzyme necessary for the degradation of glycogen. Common symptoms include skeletal and smooth muscle myopathy, ...

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

First-In-Human Study to Evaluate Safety Tolerability and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with a chaperone (AT2221). The study aims to evaluate safety, tolerability, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. The study will be conducted in 3 stages. In Stage 1, ...

Phase

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

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