Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

Pompe Disease Clinical Trial

Official title:

Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02405598
• Other study ID #: 201308031MINC
• Status: Completed
• Phase: Phase 4
• First received: March 22, 2015
• Last updated: September 18, 2016
• Start date: October 2013
• Est. completion date: June 2016

Study information

• Verified date: September 2016
• Source: National Taiwan University Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Taiwan: Bureau of Health
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: June 2016
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years and older

Eligibility

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 ß-agonists

8. under medications including diuretics, digoxin, beta-blockers, etc

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Drug:
• Salbutamol
Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot) VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	10% decrement of creatinine kinase	measure and compare serum creatinine kinase levels	6 months	Yes
Primary	Improvement in 6-minutes walk test	measure and compare 6-minutes walk test	6 months	No