Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

Status

Completed

Phases

Phase 4

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02405598

Enrollment

Registered

2015-04-01

Start date

2013-10-31

Completion date

2016-06-30

Last updated

2016-09-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe Disease

Brief summary

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

Interventions

DRUGSalbutamol

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame	Description
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

Countries

Taiwan

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026