Text Size

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

The recruitment status of this study is unknown because the information has not been verified recently.
Verified September 2008 by National Taiwan University Hospital.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
National Taiwan University Hospital
ClinicalTrials.gov Identifier:
NCT00713453
First received: July 9, 2008
Last updated: November 2, 2008
Last verified: September 2008
History of Changes
  Purpose

This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy


Condition
Pompe Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Official Title: Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by National Taiwan University Hospital:

Estimated Enrollment: 16
Study Start Date: January 2007
Estimated Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population

patients with Pompe disease, prepare or on enzyme replacement therapy

Criteria

Inclusion Criteria:

  • patients with Pompe disease, prepare or on enzyme replacement therapy
  • signed informed consent

Exclusion Criteria:

-

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00713453

Contacts
Contact: Yin-Hsiu Chien, MD 886-2-23123456 ext 7441 chienyh@ntu.edu.tw

Locations
Taiwan
National Taiwan University Hospital Recruiting
Taipei, Taiwan
Contact: Yin-Hsiu Chien, MD     886-2-23123456 ext 7441     chienyh@ntu.edu.tw    
Sub-Investigator: Whu-Liang Hwu, MD, PhD            
Principal Investigator: Yin-Hsiu Chien, MD            
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
Principal Investigator: Yin-Hsiu Chien, MD National Taiwan University Hospital
  More Information

No publications provided

Responsible Party: Yin-Hsiu Chien, MD, National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT00713453     History of Changes
Other Study ID Numbers: 200704066R
Study First Received: July 9, 2008
Last Updated: November 2, 2008
Health Authority: Taiwan: Department of Health

Keywords provided by National Taiwan University Hospital:
Pompe disease
muscle
enzyme replacement therapy

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
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
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013