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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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).


Detailed Summary:
Sponsor: Dwight Koeberl, M.D., Ph.D.

Current Primary Outcome:

  • 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


Original Primary Outcome:

  • Change in 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 AST, 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


Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome: Same as current

Information By: Duke University

Dates:
Date Received: September 11, 2013
Date Started: September 2013
Date Completion:
Last Updated: December 30, 2016
Last Verified: December 2016