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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
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

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

First Posted
March 22, 2015
Last Updated
September 18, 2016
Sponsor
National Taiwan University Hospital
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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

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