Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

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

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Key information

Conditions

Pompe Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

National Taiwan University Hospital

Taipei, Taiwan, 100

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Confirm diagnosis as Pompe disease
age 2 years or above
under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
Provide inform consent

Exclusion criteria

chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
history of seizure
history of diabetes mellitus
history of hyperthyroidism
hypokalemia
pregnant
allergy to β-agonists
under medications including diuretics, digoxin, beta-blockers, etc

Treatment and study plan

Salbutamol

Drug

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

Primary outcomes

10% decrement of creatinine kinase

Time frame: 6 months

measure and compare serum creatinine kinase levels
Improvement in 6-minutes walk test

Time frame: 6 months

measure and compare 6-minutes walk test

Sponsors and collaborators

Lead sponsor

National Taiwan University Hospital

Other

Registry information

Important dates

Study start: 2013
Primary completion: 2015
Study completion: 2016
First posted: Apr 1, 2015
Registry last updated: Sep 20, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed