Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

• Status: Completed
• Trial end date: 2016-09-02
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dwight Koeberl, M.D., Ph.D.
• Treatments: Clenbuterol

Criteria

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 ERT 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, arrythmia, cardiomyopathy)

4. Tachycardia

5. History of seizure disorder

6. Hyperthyroidism

7. Pheochromocytoma

8. Pregnancy

9. History of diabetes

10. History of hypersensitivity to beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),

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

12. Treatment for asthma in the previous 12 months.

13. 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).