Long-term follow-up for patients with Pompe disease receiving treatment
A Long-term Study for the Outcome of Pompe Disease
This study is tracking patients with Pompe disease who are getting enzyme replacement therapy to see how well the treatment works over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Sex | All |
| Sponsor | National Taiwan University Hospital Academic / other |
| Locations | 1 site (Taipei) |
| Trial ID | NCT02399748 on ClinicalTrials.gov |
What this trial studies
This study aims to prospectively follow patients diagnosed with Pompe disease who are currently undergoing or preparing to receive enzyme replacement therapy. By observing these patients over an extended period, the study seeks to gather data on the long-term outcomes and effectiveness of the treatment. The observational nature of the study allows for real-world insights into patient experiences and health changes associated with the therapy.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with Pompe disease who are currently receiving or preparing to receive enzyme replacement therapy.
Not a fit: Patients who are not diagnosed with Pompe disease or those not receiving enzyme replacement therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable information on the long-term effectiveness and safety of enzyme replacement therapy for Pompe disease patients.
How similar studies have performed: While there have been studies on enzyme replacement therapy for Pompe disease, this long-term observational approach may provide novel insights into patient outcomes over time.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy Exclusion Criteria: No
Where this trial is running
Taipei
- National Taiwan University Hospital — Taipei, Taiwan (Recruiting)
Study contacts
- Principal investigator: Yin-Hsiu Chien — National Taiwan University Hospital
- Study coordinator: Yin-Hsiu Chien
- Email: chienyh@ntu.edu.tw
- Phone: +886223123456
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.