Condition (MeSH)
Carbohydrate Metabolism, Inborn Errors
ClinicalTrials.gov lists 730 trials for Carbohydrate Metabolism, Inborn Errors or a narrower condition beneath it. Genzyme, a Sanofi Company has the most among industry and government lead sponsors, with 43.
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Trials per phase
1 more section with rows
5 of the 730 trials are tagged with Carbohydrate Metabolism, Inborn Errors itself; the rest are tagged with a narrower condition beneath it.
When did Carbohydrate Metabolism, Inborn Errors trials start? (33)
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. 2 trials are registered to start in 2027 or later, and not listed here.
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: 52
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) | 31 |
| 2025 | 25 |
| 2024 | 29 |
| 2023 | 39 |
| 2022 | 41 |
| 2021 | 44 |
| 2020 | 38 |
| 2019 | 45 |
| 2018 | 52 |
| 2017 | 32 |
Showing 10 of 33 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 38 trials starting in 2026 and about 32 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. 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 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. 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 58 trials still running at the 2026-10-09 snapshot; registered median of 27 months across 440 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Carbohydrate Metabolism, Inborn Errors trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 51 |
| Phase 1/2 | 74 |
| Phase 2 | 78 |
| Phase 2/3 | 31 |
| Phase 3 | 51 |
| Phase 4 | 43 |
| Not applicable | 115 |
| Observational | 272 |
| Expanded access | 15 |
How many Carbohydrate Metabolism, Inborn Errors trials are recruiting? (12)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 22 |
| Recruiting | 97 |
| Enrolling by invitation | 14 |
| Active, not recruiting | 42 |
| Available | 7 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Carbohydrate Metabolism, Inborn Errors trials? (9)
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 | 43 |
| BioMarin Pharmaceutical | 20 |
| Shire | 20 |
| Ultragenyx Pharmaceutical Inc | 17 |
| JCR Pharmaceuticals Co., Ltd. | 12 |
Showing 5 of 9 sponsors.
Which drugs are being tested for Carbohydrate Metabolism, Inborn Errors? (12)
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 |
| Laronidase | 15 |
| Idursulfase | 12 |
| Lamazym | 11 |
| BMN 110 | 10 |
Showing 5 of 12 drugs.
Interventions as named in the registry (3)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Idursulfase IT (1 mg) | 6 |
| Alemtuzumab s.c. | 5 |
| Observational | 5 |
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 Carbohydrate Metabolism, Inborn Errors 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 | 110 | 35 |
| Knee deformity | 76 | 5 |
| Death | 64 | 4 |
| Pyrexia | 61 | 27 |
| Poor venous access | 55 | 9 |
Showing 5 of 10 adverse events.
Which publications cover Carbohydrate Metabolism, Inborn Errors? (41)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Red blood cells as glucose carriers to the human brain: Modulation of cerebral activity by erythrocyte exchange transfusion in Glut1 deficiency (G1D). | Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism | 2023 | Yes |
| Quantitative Three-Dimensional Gait Evaluation in Patients With Glucose Transporter 1 Deficiency Syndrome. | Pediatric neurology | 2022 | Yes |
| Transition from ketogenic diet to triheptanoin in patients with GLUT1 deficiency syndrome. | Journal of neurology, neurosurgery, and psychiatry | 2020 | Yes |
| Hypomorphic SI genetic variants are associated with childhood chronic loose stools. | PloS one | 2020 | Yes |
| Long-term follow-up in an open-label trial of triheptanoin in GLUT1 deficiency syndrome: a sustained dramatic effect. | Journal of neurology, neurosurgery, and psychiatry | 2019 | Yes |
Showing 5 of 41 publications.
Sub-conditions in MeSH (13)
MeSH descendants one level down with at least one trial, most trials first.
+3 more sub-conditions
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.