Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

Phase 4

Completed

• Conditions: Pompe Disease
• Interventions: Drug: Salbutamol

• Registration Number: NCT02405598
• Lead Sponsor: National Taiwan University Hospital

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10
• Study First Submitted: 2015-03-22
• Study First Submitted QC: 2015-03-27
• Study First Posted: 2015-04-01
• Primary Completion: 2015-07
• Study Completion: 2016-06
• Status Verified: 2016-09
• Last Update Submitted: 2016-09-18
• Last Update Posted: 2016-09-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Salbutamol	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)

Primary Outcome Measures

Name	Time	Method
10% decrement of creatinine kinase	6 months	measure and compare serum creatinine kinase levels
Improvement in 6-minutes walk test	6 months	measure and compare 6-minutes walk test

Trial Locations

Locations (1)

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan