Overview

This trial is active, not recruiting.

Condition pompe disease
Treatments albuterol, placebo
Phase phase 1/phase 2
Sponsor Duke University
Start date June 2013
End date December 2016
Trial size 16 participants
Trial identifier NCT01885936, Pro00046020

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Experimental)
Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study.
albuterol
Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study.
(Placebo Comparator)
placebo

Primary Outcomes

Measure
Number of participants with Adverse Events.
time frame: 52 weeks

Secondary Outcomes

Measure
Change in forced vital capacity from pulmonary function tests at 30 weeks and 52 weeks.
time frame: Baseline and weeks 30 and 52.
Change in 6 minute walk test
time frame: Weeks 6 and 52.

Eligibility Criteria

Male or female participants at least 18 years old.

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.

Additional Information

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
Principal investigator Dwight d Koeberl, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Duke University.