A Long-term Study for the Outcome of Pompe Disease

Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.

Recruiting

Interested in participating?

Key information

Conditions

Pompe Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

National Taiwan University Hospital

Taipei, 100, Taiwan

Location status: Recruiting

Location contact

Yin-Hsiu Chien, MD, PhD

CONTACT

[email protected]

+886223123456 ext. 71937

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

Treatment and study plan

Primary outcomes

all cause morbidities

Time frame: 10 years

Study contacts

Contact information is provided by the study sponsor or research team.

Yin-Hsiu Chien

CONTACT

[email protected]

+886223123456 ext. 71937

Sponsors and collaborators

Lead sponsor

National Taiwan University Hospital

Other

Registry information

Important dates

Study start: 2007
Primary completion: 2026
Study completion: 2026
First posted: Mar 26, 2015
Registry last updated: Jul 18, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Recruiting