Condition (MeSH)
Pyruvate Metabolism, Inborn Errors
ClinicalTrials.gov lists 29 trials for Pyruvate Metabolism, Inborn Errors or a narrower condition beneath it. PTC Therapeutics has the most among industry and government lead sponsors, with 5.
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Trials per phase
When did Pyruvate Metabolism, Inborn Errors trials start? (13)
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 2
- Phase 2/3
- Phase 3
- Observational
Top of scale: 4
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) | 1 |
| 2025 | 2 |
| 2024 | 1 |
| 2022 | 3 |
| 2021 | 1 |
| 2020 | 4 |
| 2018 | 2 |
| 2015 | 4 |
| 2014 | 2 |
| 2012 | 3 |
Showing 10 of 13 earlier years.
No forecast is made for this condition: it averages 1.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials 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 Pyruvate Metabolism, Inborn Errors trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 10 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Observational | 11 |
| Expanded access | 3 |
How many Pyruvate Metabolism, Inborn Errors trials are recruiting? (11)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 9 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 1 |
| Available | 1 |
Showing 5 of 11 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Pyruvate Metabolism, Inborn Errors trials? (6)
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 |
|---|---|
| PTC Therapeutics | 5 |
| Amgen | 2 |
| Khondrion BV | 2 |
| Saol Therapeutics Inc | 2 |
| Aadi Bioscience, Inc. | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Pyruvate Metabolism, Inborn Errors? (6)
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 |
|---|---|
| EPI-743 | 5 |
| Triheptanoin | 2 |
| Dichloroacetate | 2 |
| (Cystagon) Cysteamine Bitartrate | 2 |
| KH176 | 2 |
Showing 5 of 6 drugs.
Interventions as named in the registry (6)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| off-label compassionate drug use | 1 |
| 20% mannitol | 1 |
| 4-Phenylbutyrate | 1 |
| Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] | 1 |
| TTI-0102: cysteamine-pantetheine disulfide | 1 |
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 Pyruvate 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 | 20 | 2 |
| Vomiting | 15 | 4 |
| Seizure | 14 | 2 |
| Acute respiratory failure | 12 | 2 |
| COVID-19 | 10 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Pyruvate Metabolism, Inborn Errors? (11)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency. | Blood advances | 2024 | Yes |
| Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency. | American journal of hematology | 2023 | Yes |
| The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design. | BMJ open | 2023 | Yes |
| Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency. | American journal of hematology | 2020 | Yes |
| Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. | The New England journal of medicine | 2019 | Yes |
Showing 5 of 11 publications.
Sub-conditions in MeSH (3)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.