We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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
Sponsor:
Information provided by (Responsible Party):
Hsiao Sang Chu, MD, National Taiwan University Hospital

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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)
  2. VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Locations
Layout table for location information
Taiwan
National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
Layout table for investigator information
Principal Investigator: Yin-Hsiu Chien, MD, PhD National Taiwan University Hospital
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
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:
Layout table for 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
Albuterol
 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