Condition (MeSH)
Galactosemias
ClinicalTrials.gov lists 15 trials for Galactosemias or a narrower condition beneath it. Applied Therapeutics, Inc. has the most among industry and government lead sponsors, with 3.
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Trials per phase
15 of the 15 trials are tagged with Galactosemias itself; the rest are tagged with a narrower condition beneath it.
When did Galactosemias trials start? (11)
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 2/3
- 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 |
|---|---|
| 2025 | 1 |
| 2022 | 1 |
| 2021 | 3 |
| 2019 | 2 |
| 2018 | 1 |
| 2017 | 2 |
| 2015 | 1 |
| 2014 | 1 |
| 2012 | 1 |
| 2007 | 1 |
Showing 10 of 11 earlier years.
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 15 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Galactosemias trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Phase 2 | 1 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Not applicable | 2 |
| Observational | 7 |
How many Galactosemias trials are recruiting? (4)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Active, not recruiting | 1 |
| Completed | 10 |
| Unknown status | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Galactosemias 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 |
|---|---|
| Applied Therapeutics, Inc. | 3 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
Which drugs are being tested for Galactosemias? (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 |
|---|---|
| AT-007 | 3 |
| Arginine Aspartate | 1 |
Interventions as named in the registry (3)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Ivermectin 200 µg/kg | 1 |
| Ivermectin 400 µg/kg | 1 |
| follitropin and lutropin | 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 Galactosemias trials report? (1)
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 |
|---|---|---|
| Liver Injury | 1 | 1 |
Which publications cover Galactosemias? (20)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Results of the ACTION-Galactosemia Kids Study to Evaluate the Effects of Govorestat in Pediatric Patients with Classic Galactosemia. | Journal of clinical pharmacology | 2025 | Yes |
| Feasibility of a Proactive Parent-Implemented Communication Intervention Delivered via Telepractice for Children With Classic Galactosemia. | American journal of speech-language pathology | 2022 | Yes |
| Toward Preventing Speech and Language Disorders of Known Genetic Origin: First Post-Intervention Results of Babble Boot Camp in Children With Classic Galactosemia. | American journal of speech-language pathology | 2021 | Yes |
| Toward a paradigm shift from deficit-based to proactive speech and language treatment: Randomized pilot trial of the Babble Boot Camp in infants with classic galactosemia. | F1000Research | 2019 | Yes |
| Hereditary galactosemia. | Metabolism: clinical and experimental | 2018 | Yes |
Showing 5 of 20 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Galactosemia | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Galactosemia | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Galactosemia | Prevalence at birth | 1-9 / 100 000 | United States | US | Unspecified |
| Galactosemia | Annual incidence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Galactosemia | Prevalence at birth | 1-9 / 100 000 | Europe | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Galactosemia | 352 | Exact | Orphadata Science knowledge files, Product 1 |
| Galactosemia | 352 | 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.