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About
In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated in patients with Late Onset Pompe Disease.
Enrollment
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Inclusion criteria
Exclusion criteria
Continuous invasive ventilation (via tracheostomy or endotracheal tube).
Clinically relevant illness within two weeks of enrollment including fever > 38.2 C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
Chronic heart disease (Myocardial infarction in the past 2 months, arrhythmia, cardiomyopathy).
History of seizure disorder.
History of diabetes.
Hypokalemia.
History of hyperthyroidism.
Pregnancy.
Patients on a non-standard schedule for enzyme replacement therapy; for example, weekly infusions as opposed to infusions every two weeks.
Anti-rhGAA antibody titer > 1:100,000
History of hypersensitivity to Beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent)..
The use of the following medications:
Primary purpose
Allocation
Interventional model
Masking
16 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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