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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01885936 |
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Date of registration:
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21/06/2013 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
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Scientific title:
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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 |
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Date of first enrolment:
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June 2013 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01885936 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Dwight d Koeberl, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Duke University |
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Key inclusion & exclusion criteria
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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.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: Albuterol
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Drug: Placebo
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Primary Outcome(s)
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Number of Participants With Adverse Events.
[Time Frame: 52 weeks]
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Secondary Outcome(s)
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Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.
[Time Frame: Baseline, Week 30, and Week 52]
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Change in 6 Minute Walk Test
[Time Frame: Baseline, Week 6, and Week 52]
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Secondary ID(s)
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Pro00046020
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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