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Muscle Response to Enzyme Replacement Therapy in Pompe Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2015 by National Taiwan University Hospital
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital
ClinicalTrials.gov Identifier:
NCT00713453
First received: July 9, 2008
Last updated: March 21, 2015
Last verified: March 2015
  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:


Further study details as provided by National Taiwan University Hospital:

Primary Outcome Measures:
  • all morphology changes improvement [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 16
Study Start Date: January 2007
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Contacts
Contact: Yin-Hsiu Chien, MD 886-2-23123456 ext 71937 chienyh@ntu.edu.tw

Locations
Taiwan
National Taiwan University Hospital Recruiting
Taipei, Taiwan
Contact: Yin-Hsiu Chien, MD    886-2-23123456 ext 71937    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

Responsible Party: National Taiwan University Hospital, Yin-Hsiu Chien, M.D., PhD.
ClinicalTrials.gov Identifier: NCT00713453     History of Changes
Other Study ID Numbers: 200704066R 
Study First Received: July 9, 2008
Last Updated: March 21, 2015
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 September 27, 2016