A Long Term Follow up Study in Late-onset Pompe Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by National Taiwan University Hospital
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital
ClinicalTrials.gov Identifier:
NCT00713245
First received: July 9, 2008
Last updated: December 3, 2013
Last verified: December 2013
  Purpose

This study is to observe the progression of disease in late-onset Pompe disease


Condition
Pompe Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Long Term Follow up Study in Late-onset Pompe Disease

Resource links provided by NLM:


Further study details as provided by National Taiwan University Hospital:

Primary Outcome Measures:
  • all muscle function [ Time Frame: 10 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 70
Study Start Date: May 2008
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

individuals with acid alpha glucosidase deficiency but without obvious symptoms

Criteria

Inclusion Criteria:

  • individuals with acid alpha glucosidase deficiency without obvious symptoms
  • parents signed inform consent

Exclusion Criteria:

  • individuals with acid alpha glucosidase deficiency presenting symptoms
  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: NCT00713245

Contacts
Contact: Whu-Liang Hwu, MD, PhD hwuwlntu@ntu.edu.tw

Locations
Taiwan
National Taiwan University Hospital Recruiting
Taipei, Taiwan
Contact: Yin-Hsiu Chien, MD    886-2-23123456 ext 71937      
Principal Investigator: Whu-Liang Hwu, MD, PhD         
Sub-Investigator: Yin-Hsiu Chien, MD         
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
Principal Investigator: Whu-Liang Hwu, MD, PhD National Taiwan University Hospital
  More Information

No publications provided

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT00713245     History of Changes
Other Study ID Numbers: 200804012R
Study First Received: July 9, 2008
Last Updated: December 3, 2013
Health Authority: Taiwan: Department of Health

Keywords provided by National Taiwan University Hospital:
Pompe disease
late onset

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 August 28, 2014