Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Primary Purpose
Pompe Disease
Status
Completed
Phase
Phase 4
Locations
Taiwan
Study Type
Interventional
Intervention
Salbutamol
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease
Eligibility Criteria
Inclusion Criteria:
- Confirm diagnosis as Pompe disease
- age 2 years or above
- under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
- Provide inform consent
Exclusion Criteria:
- chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
- history of seizure
- history of diabetes mellitus
- history of hyperthyroidism
- hypokalemia
- pregnant
- allergy to β-agonists
- under medications including diuretics, digoxin, beta-blockers, etc
Sites / Locations
- National Taiwan University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Salbutamol
Arm Description
age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg) age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg) age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg)
Outcomes
Primary Outcome Measures
10% decrement of creatinine kinase
measure and compare serum creatinine kinase levels
Improvement in 6-minutes walk test
measure and compare 6-minutes walk test
Secondary Outcome Measures
Full Information
NCT ID
NCT02405598
First Posted
March 22, 2015
Last Updated
September 18, 2016
Sponsor
National Taiwan University Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02405598
Brief Title
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Official Title
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Study Type
Interventional
2. Study Status
Record Verification Date
September 2016
Overall Recruitment Status
Completed
Study Start Date
October 2013 (undefined)
Primary Completion Date
July 2015 (Actual)
Study Completion Date
June 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
National Taiwan University Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Salbutamol
Arm Type
Experimental
Arm Description
age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg)
age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg)
age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg)
Intervention Type
Drug
Intervention Name(s)
Salbutamol
Intervention Description
Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot)
VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)
Primary Outcome Measure Information:
Title
10% decrement of creatinine kinase
Description
measure and compare serum creatinine kinase levels
Time Frame
6 months
Title
Improvement in 6-minutes walk test
Description
measure and compare 6-minutes walk test
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirm diagnosis as Pompe disease
age 2 years or above
under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
Provide inform consent
Exclusion Criteria:
chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
history of seizure
history of diabetes mellitus
history of hyperthyroidism
hypokalemia
pregnant
allergy to β-agonists
under medications including diuretics, digoxin, beta-blockers, etc
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien, MD, PhD
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
12. IPD Sharing Statement
Learn more about this trial
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
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