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Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy

Information source: Duke University
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Pompe Disease

Intervention: Albuterol (Drug); Placebo (Drug)

Phase: Phase 1/Phase 2

Status: Recruiting

Sponsored by: Duke University

Official(s) and/or principal investigator(s):
Dwight d Koeberl, MD, PhD, Principal Investigator, Affiliation: Duke University


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.

Clinical Details

Official title: A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome: Number of participants with Adverse Events.

Secondary outcome:

Change in forced vital capacity from pulmonary function tests at 30 weeks and 52 weeks.

Change in 6 minute walk test


Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.


Inclusion Criteria: 1. Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing, 2. Age: 18+ years at enrollment. 3. Receiving enzyme replacement therapy at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks. 4. Subjects are capable of giving written consent. Exclusion Criteria: 1. Continuous invasive ventilation (via tracheostomy or endotracheal tube). 2. 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. 3. Chronic heart disease (Myocardial infarction in the past 2 months, arrhythmia, cardiomyopathy). 4. History of seizure disorder. 5. History of diabetes. 6. Hypokalemia. 7. History of hyperthyroidism. 8. Pregnancy. 9. Patients on a non-standard schedule for enzyme replacement therapy; for example, weekly infusions as opposed to infusions every two weeks. 10. Anti-rhGAA antibody titer > 1: 100,000 11. History of hypersensitivity to Beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent).. 12. 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.

Locations and Contacts

Duke University Medical Center, Durham, North Carolina 27710, United States; Recruiting
Dwight Koeberl, MD, Phone: 919-684-2036, Email: koebe001@mc.duke.edu
Crista Walters, NP, Phone: 919-681-1945, Email: crista.walters@duke.edu
Dwight Koeberl, MD, PhD, Principal Investigator
Additional Information

Starting date: June 2013
Last updated: August 6, 2015

Page last updated: August 20, 2015

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