Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

NCT02405598 | Completed Phase 4

• 1 other identifier: 201308031MINC
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (2)

• 10% decrement of creatinine kinase

  measure and compare serum creatinine kinase levels

  6 months

• Improvement in 6-minutes walk test

  measure and compare 6-minutes walk test

  6 months

Study Arms (1)

Salbutamol

EXPERIMENTAL

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)

Drug: Salbutamol

Interventions

Salbutamol DRUG

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

Salbutamol

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• National Taiwan University Hospital: lead

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan, 100, Taiwan

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

Albuterol

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Phenethylamines; Ethylamines

Study Officials

• Yin-Hsiu Chien, MD, PhD

  National Taiwan University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: March 22, 2015
• First Posted: April 1, 2015
• Study Start: October 1, 2013
• Primary Completion: July 1, 2015
• Study Completion: June 1, 2016
• Last Updated: September 20, 2016

Record last verified: 2016-09

Locations

TW (1)