Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

September 18, 2016 updated by: Hsiao Sang Chu, MD, National Taiwan University Hospital
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Taiwan
      • Taipei, Taiwan, 100
        • National Taiwan University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
10% decrement of creatinine kinase
Time Frame: 6 months
measure and compare serum creatinine kinase levels
6 months
Improvement in 6-minutes walk test
Time Frame: 6 months
measure and compare 6-minutes walk test
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Taiwan University Hospital

Investigators

  • Principal Investigator: Yin-Hsiu Chien, MD, PhD, National Taiwan University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2013

Primary Completion (Actual)

July 1, 2015

Study Completion (Actual)

June 1, 2016

Study Registration Dates

First Submitted

March 22, 2015

First Submitted That Met QC Criteria

March 27, 2015

First Posted (Estimate)

April 1, 2015

Study Record Updates

Last Update Posted (Estimate)

September 20, 2016

Last Update Submitted That Met QC Criteria

September 18, 2016

Last Verified

September 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 201308031MINC

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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