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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Dwight Koeberl, M.D., Ph.D., Duke University Medical Center
ClinicalTrials.gov Identifier:
NCT01942590
First received: September 11, 2013
Last updated: December 30, 2016
Last verified: December 2016
  Purpose

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).


Condition Intervention Phase
Pompe Disease Drug: Clenbuterol Drug: Placebo Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Dwight Koeberl, M.D., Ph.D., Duke University Medical Center:

Primary Outcome Measures:
  • Change in creatine kinase (CK) reflecting worsening of muscle involvement [ Time Frame: Baseline, week 12, week 18, and week 52 ]
    Worsening muscle involvement, as defined by >3x increase in CK from baseline that is >2x the upper limit of normal

  • Change in aspartate aminotransferase (AST), alanine transaminase (ALT), and bilirubin representing liver toxicity [ Time Frame: Baseline, week 12, week 18, and week 52 ]
    Liver toxicity, as defined by a >3x increase in AST or ALT from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal


Secondary Outcome Measures:
  • Change 6 minute walk test [ Time Frame: Baseline, Weeks 6, 12, 18 and 52 ]
    Assess exercise tolerance in study patients; test administered by physical therapist.

  • Change in forced vital capacity in pulmonary function testing [ Time Frame: Baseline, Weeks 18 and 52 ]

Enrollment: 17
Study Start Date: September 2013
Study Completion Date: September 2016
Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Clenbuterol
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.
Drug: Clenbuterol
Placebo Comparator: Placebo Comparator Drug: Placebo

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01942590

Locations
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Dwight Koeberl, M.D., Ph.D.
Investigators
Principal Investigator: Dwight D Koeberl, MD, PhD Duke University
  More Information

Publications:
Responsible Party: Dwight Koeberl, M.D., Ph.D., Associate Professor, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01942590     History of Changes
Other Study ID Numbers: Pro00043680
R01FD004364 ( US NIH Grant/Contract Award Number )
Study First Received: September 11, 2013
Last Updated: December 30, 2016

Keywords provided by Dwight Koeberl, M.D., Ph.D., Duke University Medical Center:
Pompe disease

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Clenbuterol
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Anti-Asthmatic Agents
Respiratory System Agents
Sympathomimetics

ClinicalTrials.gov processed this record on June 23, 2017