Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

• Status: Completed
• Trial end date: 2016-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Taiwan University Hospital
• Treatments: Albuterol

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