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Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02405598
Recruitment Status : Completed
First Posted : April 1, 2015
Last Update Posted : September 20, 2016
Sponsor:
Information provided by (Responsible Party):
Hsiao Sang Chu, MD, National Taiwan University Hospital

Tracking Information
First Submitted Date  ICMJE March 22, 2015
First Posted Date  ICMJE April 1, 2015
Last Update Posted Date September 20, 2016
Study Start Date  ICMJE October 2013
Actual Primary Completion Date July 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 27, 2015)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Official Title  ICMJE Evaluation of Salbutamol as an Adjuvant Therapy for 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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pompe Disease
Intervention  ICMJE Drug: Salbutamol
  1. Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot)
  2. VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)
Study Arms  ICMJE Experimental: 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)
Intervention: Drug: Salbutamol
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 11, 2016)		 14
Original Estimated Enrollment  ICMJE
 (submitted: March 27, 2015)		 20
Actual Study Completion Date  ICMJE June 2016
Actual Primary Completion Date July 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Taiwan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02405598
Other Study ID Numbers  ICMJE 201308031MINC
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Hsiao Sang Chu, MD, National Taiwan University Hospital
Original Responsible Party National Taiwan University Hospital, MD, PhD
Current Study Sponsor  ICMJE National Taiwan University Hospital
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Yin-Hsiu Chien, MD, PhD National Taiwan University Hospital
PRS Account National Taiwan University Hospital
Verification Date September 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP