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Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02405598
Recruitment Status : Completed
First Posted : April 1, 2015
Last Update Posted : September 20, 2016
Information provided by (Responsible Party):
Hsiao Sang Chu, MD, National Taiwan University Hospital

Brief Summary:
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy

Condition or disease Intervention/treatment Phase
Pompe Disease Drug: Salbutamol Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Study Start Date : October 2013
Actual Primary Completion Date : July 2015
Actual Study Completion Date : June 2016

Arm Intervention/treatment
Experimental: Salbutamol
  1. age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg)
  2. age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg)
  3. age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg)
Drug: Salbutamol
  1. Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot)

Primary Outcome Measures :
  1. 10% decrement of creatinine kinase [ Time Frame: 6 months ]
    measure and compare serum creatinine kinase levels

  2. Improvement in 6-minutes walk test [ Time Frame: 6 months ]
    measure and compare 6-minutes walk test

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Confirm diagnosis as Pompe disease
  2. age 2 years or above
  3. under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
  4. Provide inform consent

Exclusion Criteria:

  1. chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
  2. history of seizure
  3. history of diabetes mellitus
  4. history of hyperthyroidism
  5. hypokalemia
  6. pregnant
  7. allergy to β-agonists
  8. under medications including diuretics, digoxin, beta-blockers, etc

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02405598

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National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
National Taiwan University Hospital
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Principal Investigator: Yin-Hsiu Chien, MD, PhD National Taiwan University Hospital
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Responsible Party: Hsiao Sang Chu, MD, MD, PhD, National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT02405598    
Other Study ID Numbers: 201308031MINC
First Posted: April 1, 2015    Key Record Dates
Last Update Posted: September 20, 2016
Last Verified: September 2016
Additional relevant MeSH terms:
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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
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Tocolytic Agents
Reproductive Control Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action