This trial has been completed.

Condition pompe disease
Treatments clenbuterol, placebo
Phase phase 1/phase 2
Sponsor Dwight Koeberl, M.D., Ph.D.
Start date September 2013
End date September 2016
Trial size 17 participants
Trial identifier NCT01942590, Pro00043680, R01FD004364


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).

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Initially, 40 mcg each morning for one week. Then, increase to 40 mcg BID for the next 5 weeks. If tolerated, will increase to 80 mcg in the morning and 40 mcg in the evening for one week. Then, last dose increase will be 80 mcg BID until week 52.
(Placebo Comparator)

Primary Outcomes

Change in creatine kinase (CK) reflecting worsening of muscle involvement
time frame: Baseline, week 12, week 18, and week 52
Change in aspartate aminotransferase (AST), alanine transaminase (ALT), and bilirubin representing liver toxicity
time frame: Baseline, week 12, week 18, and week 52

Secondary Outcomes

Change 6 minute walk test
time frame: Baseline, Weeks 6, 12, 18 and 52
Change in forced vital capacity in pulmonary function testing
time frame: Baseline, Weeks 18 and 52

Eligibility Criteria

Male or female participants at least 18 years old.

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).

Additional Information

Official title A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
Principal investigator Dwight D Koeberl, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in December 2016.
Information provided to ClinicalTrials.gov by Duke University.