Status:
COMPLETED
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Lead Sponsor:
Duke University
Conditions:
Pompe Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
June 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 16 2016
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT01885936
Start Date
June 1 2013
End Date
December 16 2016
Last Update
July 15 2019
Active Locations (1)
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1
Duke University Medical Center
Durham, North Carolina, United States, 27710