Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Pompe Disease
In brief
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.
Key facts
- Study ID
- NCT01885936
- Run by
- Duke University
- People needed
- 16
- Starts
- 2013-06-01
- Expected to finish
- 2016-12-16
- Last updated by the study team
- 2019-07-15
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
- Age: 18+ years at enrollment.
- Receiving enzyme replacement therapy at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks.
- Subjects are capable of giving written consent.
You may not qualify if…
- 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:
- diuretics (water pill);
- digoxin (digitalis, Lanoxin);
- beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);
- tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor);
- Monoamine oxidase inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
- bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetharine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer) within 12 weeks prior to enrollment.
Where it is running
- Duke University Medical Center — Durham, North Carolina, United States
Full record on ClinicalTrials.gov
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