Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Pompe Disease
In brief
Funding Source- FDA OOPD The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT).
Key facts
- Study ID
- NCT01942590
- Run by
- Dwight Koeberl, M.D., Ph.D.
- People needed
- 17
- Starts
- 2013-09-01
- Expected to finish
- 2016-09-02
- Last updated by the study team
- 2019-07-02
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
- Age: 18+ years at enrollment,
- Receiving ERT at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks,
- Subjects are capable of giving written consent.
You may not qualify if…
- 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, arrythmia, cardiomyopathy)
- Tachycardia
- History of seizure disorder
- Hyperthyroidism
- Pheochromocytoma
- Pregnancy
- History of diabetes
- History of hypersensitivity to beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),
- Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed to infusions every two weeks.
- Treatment for asthma in the previous 12 months.
- 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 inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
- other bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).
Where it is running
- Duke University Medical Center — Durham, North Carolina, United States
Full record on ClinicalTrials.gov
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