Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01859624
• Date of registration: 08/05/2013
• Prospective Registration: No
• Primary sponsor: Duke University
• Public title: Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
• Scientific title: A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement Therapy
• Date of first enrolment: June 2012
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01859624
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Dwight D Koeberl, MD, PhD
• Affiliation: Duke University

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Diagnosis of Pompe disease by blood acid alpha-glucosidase (GAA) assay and GAA gene
sequencing

2. Age: 18+ years at enrollment

Exclusion Criteria:

1. Continuous invasive ventilation (via tracheostomy or endotracheal tube)

2. Clinically relevant illness within two weeks of enrollment including fever > 38.2o C,
vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative
to new therapy.

3. Chronic heart disease (Myocardial infarction, arrythmia, cardiomyopathy)

4. History of seizure disorder

5. Hypothyroidism

6. Pregnancy/Breast Feeding [Women of childbearing potential must have a negative urine
pregnancy test at each study visit. In addition, at Screening/Baseline women of
childbearing potential must have been using a medically acceptable contraceptive for
at least 3 months prior to study enrollment OR the subject a) has a regular menstrual
cycle, b) Day 1 (onset of menses) for the current cycle is known, and c) the urine
pregnancy test can be administered within the first two weeks of the current cycle
(between Days 1 and 14)]. The urine pregnancy test will be administered and
interpreted by Stephanie Dearmey, Physician Assistant (PA-C), who has completed
training from the Department of Obstetrics and Gynecology. Mrs. Dearmey will use a
commercially available test kit specified by the point-of-care testing policies. If
these criteria for urine pregnancy testing are not met at the Screening/Baseline
visit, then a blood pregnancy test will be done.

7. Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed
to infusions every two weeks.

The use of the following concommitant meds is prohibited during the study:

- 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 (MAO) inhibitors such as isocarboxazid (Marplan), phenelzine
(Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine
(Parnate); or

- other bronchodilators such as levalbuterol (Xopenex), bitolterol (Tornalate),
pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent),
isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol
(Isuprel Mistometer).



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Drug: Albuterol

Primary Outcome(s)

Number of Participants with Adverse Events [Time Frame: 24 weeks]

Secondary Outcome(s)

Change in 6 minute walk test in 6 months. [Time Frame: Baseline and 24 weeks]

Change in forced vital capacity at 3 months [Time Frame: Baseline and week 12]

Secondary ID(s)

Pro00034177

Source(s) of Monetary Support

Please refer to primary and secondary sponsors