Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
| Status: | Completed |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 4/21/2016 |
| Start Date: | June 2012 |
| End Date: | April 2013 |
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
Albuterol is a drug approved by the US Food and Drug Administration (FDA) for treating
breathing problems such as asthma. Studies have shown that albuterol may be beneficial in
improving muscle function in people with late-onset Pompe disease. The purpose of this study
is to evaluate whether albuterol is safe and effective for improving muscle function in
people with late-onset Pompe disease, whether or not they are receiving enzyme replacement
therapy (ERT). For this study, albuterol is considered an investigational drug. The word
"investigational" means albuterol is not approved by the FDA for individuals with late-onset
Pompe disease.
breathing problems such as asthma. Studies have shown that albuterol may be beneficial in
improving muscle function in people with late-onset Pompe disease. The purpose of this study
is to evaluate whether albuterol is safe and effective for improving muscle function in
people with late-onset Pompe disease, whether or not they are receiving enzyme replacement
therapy (ERT). For this study, albuterol is considered an investigational drug. The word
"investigational" means albuterol is not approved by the FDA for individuals with late-onset
Pompe disease.
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).
We found this trial at
1
site
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
Click here to add this to my saved trials
