Condition (MeSH)
Glycogen Storage Disease Type II
ClinicalTrials.gov lists 146 trials for Glycogen Storage Disease Type II or a narrower condition beneath it. Genzyme, a Sanofi Company has the most among industry and government lead sponsors, with 33.
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Trials per phase (log scale)
1 more section with rows
146 of the 146 trials are tagged with Glycogen Storage Disease Type II itself; the rest are tagged with a narrower condition beneath it.
When did Glycogen Storage Disease Type II trials start? (25)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
- Other (Expanded access)
Top of scale: 10
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 6 |
| 2025 | 4 |
| 2024 | 8 |
| 2023 | 7 |
| 2022 | 7 |
| 2021 | 10 |
| 2020 | 5 |
| 2019 | 5 |
| 2018 | 6 |
| 2017 | 6 |
Showing 10 of 25 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 8 trials starting in 2026 and about 6 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 10 trials still running at the 2026-10-09 snapshot; registered median of 32 months across 79 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Glycogen Storage Disease Type II trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 9 |
| Phase 1/2 | 13 |
| Phase 2 | 12 |
| Phase 2/3 | 2 |
| Phase 3 | 6 |
| Phase 4 | 19 |
| Not applicable | 20 |
| Observational | 62 |
| Expanded access | 3 |
How many Glycogen Storage Disease Type II trials are recruiting? (10)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 5 |
| Recruiting | 20 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 12 |
| Available | 1 |
Showing 5 of 10 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Glycogen Storage Disease Type II trials? (8)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Genzyme, a Sanofi Company | 33 |
| Amicus Therapeutics | 9 |
| Sanofi | 4 |
| Shionogi | 3 |
| AskBio Inc | 2 |
Showing 5 of 8 sponsors.
Which drugs are being tested for Glycogen Storage Disease Type II? (9)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| ALGLUCOSIDASE ALFA | 28 |
| Avalglucosidase Alfa | 7 |
| RITUXIMAB | 4 |
| Miglustat | 4 |
| Cipaglucosidase Alfa | 3 |
Showing 5 of 9 drugs.
Interventions as named in the registry (6)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Observational | 4 |
| AT2221 | 3 |
| S-606001 | 3 |
| ATB200 | 3 |
| HD- Methotrexate (MTX) | 2 |
Showing 5 of 6 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Glycogen Storage Disease Type II trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pneumonia | 49 | 11 |
| Pyrexia | 18 | 6 |
| Respiratory distress | 18 | 2 |
| Supraventricular tachycardia | 12 | 6 |
| Hypersensitivity | 11 | 4 |
Showing 5 of 10 adverse events.
Which publications cover Glycogen Storage Disease Type II? (141)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL. | Muscle & nerve | 2025 | Yes |
| The Mini-COMET Clinical Trial: Safety and Efficacy of Avalglucosidase Alfa after 97 Weeks of Treatment in Children with Infantile-Onset Pompe Disease Previously Treated with Alglucosidase Alfa. | The Journal of pediatrics | 2025 | Yes |
| Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial. | Journal of neurology | 2025 | Yes |
| An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease. | Advances in therapy | 2025 | Yes |
| Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02). | Journal of neurology | 2024 | Yes |
Showing 5 of 141 publications.
Prevalence (28)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Glycogen storage disease due to acid maltase deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Glycogen storage disease due to acid maltase deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Glycogen storage disease due to acid maltase deficiency | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Glycogen storage disease due to acid maltase deficiency | Prevalence at birth | 1-9 / 1 000 000 | Australia | Not stated | Exact |
| Glycogen storage disease due to acid maltase deficiency | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Exact |
Showing 5 of 28 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Glycogen storage disease due to acid maltase deficiency | 365 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Glycogen storage disease due to acid maltase deficiency | 365 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.