Phase 2 N=13 Randomized Quadruple-blind Treatment

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

Pompe Disease

Bottom Line

View on ClinicalTrials.gov: NCT01942590 ↗

Enrolled (actual)

Serious AEs

7.7%

Results posted

Oct 2017

Primary outcome: Primary: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement — 1; 0 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Clenbuterol (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Dwight Koeberl, M.D., Ph.D.
Primary completion: Sep 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement	1; 0	—
PRIMARY Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity	0; 0	—
SECONDARY Change in 6 Minute Walk Test	16.42; -18.13	0.0816
SECONDARY Change in 6 Minute Walk Test	16.42; -18.13	0.0816
SECONDARY Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing	-5.738; 7.775	0.233
SECONDARY Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing	-5.738; 7.775	0.233
SECONDARY Change in Urinary Glc4 Biomarker	-1.1; -1.667	0.161
SECONDARY Change in Urinary Glc4 Biomarker	-1.1; -1.667	0.161
SECONDARY GSGC (Gait, Stairs, Gowers, Arising From a Chair.)	17; 7.5; 15.14; 6.5; 13.8; 6.5	0.01 sig
SECONDARY Quick Motor Function Test (QMFT)	35; 53.75; 40.6; 54.75; 46.5; 56.25	0.006 sig
SECONDARY Late-Life Function and Disability Instrument (LLFDI)	103.75; 106.7; 112.5	0.3639
SECONDARY Predicted Maximum Inspiration Pressure (MIP)	56.3; 96.8; 47.4; 83.8; 68.5; 104.6	0.268
SECONDARY Maximum Expiratory Pressure (MEP)	40.4; 62.8; 40; 83.3; 53.9; 49.2	0.479

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).

Eligibility Criteria

Inclusion Criteria

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.

Exclusion Criteria

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).

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Data sourced from ClinicalTrials.gov (NCT01942590). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.