Phase 2
N=13
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Pompe Disease
Bottom Line
View on ClinicalTrials.gov: NCT01885936 ↗Enrolled (actual)
13
Serious AEs
0.0%
Results posted
Feb 2018
Primary outcome: Primary: Number of Participants With Adverse Events. — 5; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Albuterol (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Duke University
- Primary completion
- Dec 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events. |
5; 5 | — |
| SECONDARY Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks. |
-0.2; 0.4; -1.3; 3.0 | — |
| SECONDARY Change in 6 Minute Walk Test |
24.0; 32.0; 43.6; 13.6 | — |
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 albuterol was well-tolerated in patients with Late Onset Pompe Disease.
Eligibility Criteria
Inclusion Criteria
- 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 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:
- 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.
Data sourced from ClinicalTrials.gov (NCT01885936). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.