Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Long-term Study for the Outcome of 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02399748
Recruitment Status : Recruiting
First Posted : March 26, 2015
Last Update Posted : July 18, 2017
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

Brief Summary:
Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.

Condition or disease
Pompe Disease

Layout table for study information
Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Long-term Study for the Outcome of Pompe Disease
Study Start Date : April 2007
Estimated Primary Completion Date : December 2026
Estimated Study Completion Date : December 2026





Primary Outcome Measures :
  1. all cause morbidities [ Time Frame: 10 years ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients of Pompe disease who under enzyme replacement therapy
Criteria

Inclusion Criteria:

Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy

Exclusion Criteria:

No


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02399748


Contacts
Layout table for location contacts
Contact: Yin-Hsiu Chien +886223123456 ext 71937 chienyh@ntu.edu.tw

Locations
Layout table for location information
Taiwan
National Taiwan University Hospital Recruiting
Taipei, Taiwan, 100
Contact: Yin-Hsiu Chien, MD, PhD    +886223123456 ext 71937    chienyh@ntu.edu.tw   
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
Layout table for investigator information
Principal Investigator: Yin-Hsiu Chien National Taiwan University Hospital

Layout table for additonal information
Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT02399748     History of Changes
Other Study ID Numbers: 200703045R
First Posted: March 26, 2015    Key Record Dates
Last Update Posted: July 18, 2017
Last Verified: July 2017
Additional relevant MeSH terms:
Layout table for 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
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors