An observational study in Pompe Disease, sponsored by National Taiwan University Hospital. Recruiting at 1 site in Taiwan. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by National Taiwan University Hospital · Observational
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Among 25 studies in Glycogen Storage Disease Type II with a site in Taiwan — 4 open to participants now, 8 under way, 2 of them phase 3. Glycogen Storage Disease Type II · Taiwan.
Among the 25 studies in Glycogen Storage Disease Type II with a site in Taiwan, this one is the 7th largest by planned enrollment of the 10 observational ones that give a count, is one of 2 that started in 2023, is one of 10 observational ones, and was the 25th registered.
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
Browse Glycogen Storage Disease Type II studies →4 of the 25 studies in Glycogen Storage Disease Type II with a site in Taiwan are open now. Recruiting first, the newest first:
Amicus Therapeutics · Sites in Taiwan, United States, Germany and 3 more countries
Ages up to 17
National Taiwan University Hospital · Sites in Taiwan
Genzyme, a Sanofi Company · Sites in Taiwan, United States, Argentina and 41 more countries
A study can be open while its sites in Taiwan have not opened yet or have stopped. Only those recruiting at a site in Taiwan →
Of the 23 Glycogen Storage Disease Type II studies recruiting now, 16 (70%) set no upper age limit and 6 (26%) stop at 75 or younger; 15 take children under 18; none take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
25 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 2 3 · Phase 3 7 · Phase 4 4
First in 2003; this study’s year is marked. 2 started in 2023.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 40 is below the median of 50 across 64 observational studies indexed under Glycogen Storage Disease Type II.
Median enrollment 20 across the 14 interventional studies, 60 across the 10 observational. Of the 10 that completed or were terminated, 4 (40%) have results posted.
This tab as a report to print or save as PDF → With an account.
14 of 25 have an industry lead sponsor. The 7 most frequent:
National Taiwan University Hospital is the lead sponsor of 2,617 studies on the registry; 578 are open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 2 (18%) have results posted.
National Taiwan University Hospital has 9 studies filed under Glycogen Storage Disease Type II — fewer than ten, so there is no comparison to draw.
3 of the studies in Glycogen Storage Disease Type II with a site in Taiwan that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04808505Phase 3Amicus TherapeuticsA Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | 2027-07 |
| NCT02399748National Taiwan University HospitalA Long-term Study for the Outcome of Pompe Disease | 2026-12 |
| NCT06150820Astellas Gene TherapiesA Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease | 2027-06-30 |
Add all 3 to your calendar (.ics) → With an account.
Among the 25 studies in Glycogen Storage Disease Type II with a site in Taiwan, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.
Clinical characteristics of Pompe patients receiving avalglucosidase alfa will be collected and analyzed. Adverse events occurring during avalglucosidase alfa treatment will be collected and described. Urinary Glc4/Hex4 is the primary endpoints for effectiveness analysis of avalglucosidase alfa. Other assessment items include creatine kinase, motor function (Quick Motor Function Test) and respiratory function (forced vital capacity). The evolution of those assessment items after avalglucosidase alfa treatment will be descriptively analyzed and compared to baseline assessments (i.e., the time before initiating avalglucosidase alfa and/or one-year preceding avalglucosidase alfa initiation) at the individual patient level depending on prior duration of ERT at time of avalglucosidase alfa switch.
Pompe Patients receiving Avalglucosidase alfa
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
-Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Urinary Glc4/Hex4
the primary endpoints for effectiveness analysis of avalglucosidase alfa. Urinary Glc4/Hex4 is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD
Time frame: 3years
Creatine kinase
Serum CK is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD (Kishnani et al., 2006; Li et al., 2021; Young et al., 2009).
Time frame: 3years
motor function (Quick Motor Function Test)
QMFT score is strongly correlated with proximal muscle strength, and significantly differentiated between patients with different levels of mobility (van Capelle et al., 2012), muscle function will be assessed by QMFT annually.
Time frame: 3 years
respiratory function (forced vital capacity).
Pulmonary function will be assessed by forced vital capacity. For infants, pulmonary function tests are difficult to administer and therefore data might be limited.
Time frame: 3 years
Started Oct 2023 (actual). Primary completion is estimated for Oct 2036, 156 months after the start.
Of 29 observational studies under Glycogen Storage Disease Type II, half reached primary completion within 108 months of starting (the median), counting those still running; a quarter within 33. This study's planned 156 months is above that.
How these figures are worked out, and which studies are left out of them.
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Glycogen Storage Disease Type II →
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