Condition (MeSH)
Glycogen Storage Disease Type I
ClinicalTrials.gov lists 16 trials for Glycogen Storage Disease Type I or a narrower condition beneath it. Ultragenyx Pharmaceutical Inc has the most among industry and government lead sponsors, with 4.
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Trials per phase
16 of the 16 trials are tagged with Glycogen Storage Disease Type I itself; the rest are tagged with a narrower condition beneath it.
When did Glycogen Storage Disease Type I trials start? (10)
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 3
- Not applicable
- Observational
Top of scale: 3
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) | 3 |
| 2025 | 1 |
| 2024 | 1 |
| 2022 | 1 |
| 2021 | 2 |
| 2020 | 1 |
| 2019 | 2 |
| 2018 | 3 |
| 2017 | 1 |
| 2013 | 1 |
No forecast is made for this condition: it averages 1.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Glycogen Storage Disease Type I trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 2 |
| Phase 2 | 2 |
| Phase 3 | 1 |
| Not applicable | 3 |
| Observational | 7 |
How many Glycogen Storage Disease Type I trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 4 |
| Active, not recruiting | 2 |
| Completed | 6 |
| Withdrawn | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Glycogen Storage Disease Type I trials? (3)
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 | 4 |
| CENTOGENE GmbH Rostock | 1 |
| ModernaTX, Inc. | 1 |
Which drugs are being tested for Glycogen Storage Disease Type I? (2)
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 |
| PREDNISOLONE | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Empagliflozine | 2 |
| steroid regimen | 1 |
| mRNA-3745 | 1 |
| fructose | 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 serious adverse events do Glycogen Storage Disease Type I 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 |
|---|---|---|
| Lactic Acidosis | 4 | 2 |
| Nephrolithiasis | 4 | 2 |
| Cellulitis | 3 | 2 |
| Abdominal Pain | 2 | 1 |
| Covid-19 | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Glycogen Storage Disease Type I? (23)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa). | Journal of inherited metabolic disease | 2025 | Yes |
| A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia. | Orphanet journal of rare diseases | 2021 | Yes |
| Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia. | Biochemical and biophysical research communications | 2020 | Yes |
| Neutropenia in glycogen storage disease Ib: outcomes for patients treated with granulocyte colony-stimulating factor. | Current opinion in hematology | 2019 | Yes |
| An evolutionary approach to optimizing glucose-6-phosphatase-α enzymatic activity for gene therapy of glycogen storage disease type Ia. | Journal of inherited metabolic disease | 2019 | Yes |
Showing 5 of 23 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 glucose-6-phosphatase deficiency | Prevalence at birth | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Glycogen storage disease due to glucose-6-phosphatase deficiency | Prevalence at birth | 1-9 / 100 000 | Worldwide | Not stated | Unspecified |
| Glycogen storage disease due to glucose-6-phosphatase deficiency | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Glycogen storage disease due to glucose-6-phosphatase deficiency | Point prevalence | Unknown | Worldwide | Not stated | Unspecified |
| Glycogen storage disease due to glucose-6-phosphatase deficiency | Prevalence at birth | 1-9 / 100 000 | 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 glucose-6-phosphatase deficiency | 364 | Exact | Orphadata Science knowledge files, Product 1 |
| Glycogen storage disease due to glucose-6-phosphatase deficiency | 364 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.