Condition (MeSH)
Glycogen Storage Disease Type III
ClinicalTrials.gov lists 11 trials for Glycogen Storage Disease Type III or a narrower condition beneath it. Ultragenyx Pharmaceutical Inc has the most among industry and government lead sponsors, with 3.
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Trials per phase
11 of the 11 trials are tagged with Glycogen Storage Disease Type III itself; the rest are tagged with a narrower condition beneath it.
When did Glycogen Storage Disease Type III trials start? (9)
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 1/2
- Phase 2
- Not applicable
- Observational
Top of scale: 2
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 |
|---|---|
| 2024 | 1 |
| 2022 | 1 |
| 2021 | 1 |
| 2020 | 1 |
| 2018 | 2 |
| 2016 | 1 |
| 2015 | 1 |
| 2013 | 2 |
| 2011 | 1 |
No forecast is made for this condition: it averages 0.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 11 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Glycogen Storage Disease Type III trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1/2 | 1 |
| Phase 2 | 1 |
| Not applicable | 3 |
| Observational | 6 |
How many Glycogen Storage Disease Type III trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Completed | 4 |
| Terminated | 2 |
| Withdrawn | 1 |
| Unknown status | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Glycogen Storage Disease Type III trials? (2)
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 |
|---|---|
| Ultragenyx Pharmaceutical Inc | 3 |
| CENTOGENE GmbH Rostock | 1 |
Which drugs are being tested for Glycogen Storage Disease Type III? (1)
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 |
|---|---|
| Triheptanoin | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| UX053 | 1 |
| Antipyretic | 1 |
| H1 Blocker | 1 |
| H2 Blocker | 1 |
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 publications cover Glycogen Storage Disease Type III? (9)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Effects of acute nutritional ketosis during exercise in adults with glycogen storage disease type IIIa are phenotype-specific: An investigator-initiated, randomized, crossover study. | Journal of inherited metabolic disease | 2021 | Yes |
| Muscle Ultrasound in Patients with Glycogen Storage Disease Types I and III. | Ultrasound in medicine & biology | 2016 | Yes |
| Glycogen storage disease type III: diagnosis, genotype, management, clinical course and outcome. | Journal of inherited metabolic disease | 2016 | Yes |
| Skeletal muscle metabolism is impaired during exercise in glycogen storage disease type III. | Neurology | 2015 | Yes |
| Exercise intolerance in Glycogen Storage Disease Type III: weakness or energy deficiency? | Molecular genetics and metabolism | 2013 | Yes |
Showing 5 of 9 publications.
Prevalence (6)
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 glycogen debranching enzyme deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Glycogen storage disease due to glycogen debranching enzyme deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Glycogen storage disease due to glycogen debranching enzyme deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Unspecified |
| Glycogen storage disease due to glycogen debranching enzyme deficiency | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Glycogen storage disease due to glycogen debranching enzyme deficiency | Point prevalence | Unknown | Worldwide | Not stated | Exact |
Showing 5 of 6 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 glycogen debranching enzyme deficiency | 366 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Glycogen storage disease due to glycogen debranching enzyme deficiency | 366 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.