Actively Recruiting
Led by National Taiwan University Hospital · Updated on 2017-07-18
50
Participants Needed
1
Research Sites
N/A
Total Duration
This observational study follows patients diagnosed with Pompe disease who are receiving or preparing to receive enzyme replacement therapy. The research aims to monitor long-term outcomes and the overall health of these patients over an extended period, providing valuable information about the disease progression and treatment experience. Participants will be observed prospectively without any investigational treatments assigned by the study. All enrolled individuals are those already receiving enzyme replacement therapy or preparing to begin it, and the study collects data on their health and treatment outcomes over time. During the study, patients will be regularly monitored for all-cause morbidities over a 10-year period. Researchers will collect health information to understand the long-term effects of enzyme replacement therapy in Pompe disease. Participation involves ongoing health assessments, and the study extends until December 2026, providing extensive long-term outcome data.
CONDITIONS
A Long-term Study for the Outcome of Pompe Disease
You may qualify if you...
You will not qualify if you...
Complete this quick 3-step screening to check your eligibility
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 10 years
Participants are observed over an extended period to assess long-term outcomes of Pompe disease.
Periodic visits over the study duration
Total: 1 location
1
National Taiwan University Hospital
Taipei, Taiwan, 100
Actively Recruiting
Y
Yin-Hsiu Chien
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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Yu-Kang Hsu, Yin-Hsiu Chien, Steven Shinn-Forng Peng...
https://pubmed.ncbi.nlm.nih.gov/36399131