Pompe disease long-term monitoring with avalglucosidase alfa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational program collects clinical data from Pompe patients who switch to avalglucosidase alfa, tracking adverse events and several effectiveness measures. Primary focus is urinary Glc4/Hex4 as an indicator of treatment effect, with additional data on creatine kinase, motor function via the Quick Motor Function Test, and forced vital capacity for respiratory function. The evolution of these measures will be described and compared to each patient’s baseline before initiation or prior to a year before initiation, enabling descriptive analyses at the individual level. The study is conducted at National Taiwan University Hospital, led by NTUH with no listed collaborators.

Why it matters

Who can join: the full rules

Inclusion criteria:

-Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient

Exclusion criteria:

* Having severe allergic reaction to avalglucosidase alfa.
* Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if \<18 years of age

Where it's running

• National Taiwan University Hospital
  Taipei, Taiwan, Taiwan Recruiting

Study contacts

• Principal investigator: Yin-Hsiu Chien, MD, PhD — National Taiwan University Hospital Taipei, Taiwan, 100
• Study coordinator: Yin-Hsiu Chien, MD, PhD
• Email: chienyh@ntu.edu.tw
• Phone: +886223123456

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.