Condition (MeSH)
Propionic Acidemia
ClinicalTrials.gov lists 23 trials for Propionic Acidemia or a narrower condition beneath it. ModernaTX, Inc. has the most among industry and government lead sponsors, with 4.
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Trials per phase
23 of the 23 trials are tagged with Propionic Acidemia itself; the rest are tagged with a narrower condition beneath it.
When did Propionic Acidemia trials start? (14)
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: 6
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 |
| 2024 | 1 |
| 2023 | 1 |
| 2022 | 2 |
| 2021 | 6 |
| 2019 | 2 |
| 2018 | 2 |
| 2017 | 1 |
| 2016 | 1 |
| 2015 | 1 |
Showing 10 of 14 earlier years.
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: 2 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 Propionic Acidemia trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 2 |
| Phase 2 | 5 |
| Phase 3 | 1 |
| Not applicable | 2 |
| Observational | 10 |
How many Propionic Acidemia 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 | 7 |
| Active, not recruiting | 1 |
| Completed | 9 |
| Terminated | 4 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Propionic Acidemia trials? (5)
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 |
|---|---|
| ModernaTX, Inc. | 4 |
| HemoShear Therapeutics | 2 |
| RECORDATI GROUP | 2 |
| CoA Therapeutics, Inc., a BridgeBio company | 1 |
| National Human Genome Research Institute (NHGRI) | 1 |
Which drugs are being tested for Propionic Acidemia? (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 |
|---|---|
| Carglumic Acid | 5 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| mRNA-3927 | 2 |
| N-carbamylglutamate | 2 |
| Standard of Care Therapy | 1 |
| BBP-671 | 1 |
| AAVrh10-PCCA low dose | 1 |
Showing 5 of 12 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 Propionic Acidemia trials report? (6)
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 |
|---|---|---|
| Gastrointestinal Disorders | 9 | 1 |
| Infections and Infestations | 6 | 1 |
| Nervous System | 3 | 1 |
| Injury | 2 | 1 |
| Metabolic and Nutritional | 2 | 1 |
Showing 5 of 6 adverse events.
Which publications cover Propionic Acidemia? (14)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Breath biopsy in inborn errors of metabolism: A proof-of-principle study in propionic acidemia. | Molecular genetics and metabolism | 2025 | Yes |
| Real-World Experience of Carglumic Acid for Methylmalonic and Propionic Acidurias: An Interim Analysis of the Multicentre Observational PROTECT Study. | Drugs in R&D | 2024 | Yes |
| Rapid Targeted Sequencing Using Dried Blood Spot Samples for Patients With Suspected Actionable Genetic Diseases. | Annals of laboratory medicine | 2023 | Yes |
| Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia. | Nucleic acid therapeutics | 2023 | Yes |
| Long-term use of carglumic acid in methylmalonic aciduria, propionic aciduria and isovaleric aciduria in Italy: a qualitative survey. | European review for medical and pharmacological sciences | 2022 | Yes |
Showing 5 of 14 publications.
Prevalence (27)
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 |
|---|---|---|---|---|---|
| Propionic acidemia | Prevalence at birth | 1-9 / 1 000 000 | Italy | Not stated | Exact |
| Propionic acidemia | Annual incidence | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Propionic acidemia | Annual incidence | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Propionic acidemia | Prevalence at birth | >1 / 1000 | Canada | Not stated | Unspecified |
| Propionic acidemia | Prevalence at birth | 1-9 / 1 000 000 | Italy | Not stated | Exact |
Showing 5 of 27 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Propionic acidemia | 35 | Exact | Orphadata Science knowledge files, Product 1 |
| Propionic acidemia | 35 | 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.