Condition (MeSH)
Wolfram Syndrome
ClinicalTrials.gov lists 12 trials for Wolfram Syndrome or a narrower condition beneath it. Amylyx Pharmaceuticals Inc. has the most among industry and government lead sponsors, with 1.
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Trials per phase
12 of the 12 trials are tagged with Wolfram Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Wolfram Syndrome 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 1/2
- Phase 2
- Not applicable
- Observational
Top of scale: 2
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 | 1 |
| 2024 | 1 |
| 2023 | 1 |
| 2021 | 1 |
| 2019 | 1 |
| 2018 | 2 |
| 2017 | 1 |
| 2012 | 1 |
| 2011 | 2 |
No forecast is made for this condition: it averages 1.0 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 Wolfram Syndrome trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1/2 | 1 |
| Phase 2 | 4 |
| Not applicable | 1 |
| Observational | 6 |
How many Wolfram Syndrome 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 | 2 |
| Recruiting | 2 |
| Active, not recruiting | 1 |
| Completed | 4 |
| Withdrawn | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Wolfram Syndrome trials? (1)
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 |
|---|---|
| Amylyx Pharmaceuticals Inc. | 1 |
Which drugs are being tested for Wolfram Syndrome? (3)
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 |
|---|---|
| Exenatide | 1 |
| Dantrolene | 1 |
| TIRZEPATIDE | 1 |
Interventions as named in the registry (5)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Retrospective case-control analysis | 1 |
| Sodium Valproate 200Mg E/C Tablet | 1 |
| AMX0035 | 1 |
| Depakine | 1 |
| None AHT | 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 publications cover Wolfram Syndrome? (9)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial. | BMJ open | 2025 | Yes |
| A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome. | JCI insight | 2021 | Yes |
| Wolfram Syndrome: Diagnosis, Management, and Treatment. | Current diabetes reports | 2016 | Yes |
| Selective cognitive and psychiatric manifestations in Wolfram Syndrome. | Orphanet journal of rare diseases | 2015 | Yes |
| β-cell dysfunction due to increased ER stress in a stem cell model of Wolfram syndrome. | Diabetes | 2014 | Yes |
Showing 5 of 9 publications.
Prevalence (18)
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 |
|---|---|---|---|---|---|
| Wolfram syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Wolfram syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Wolfram syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Wolfram syndrome | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Wolfram syndrome | Point prevalence | 1-9 / 1 000 000 | Japan | Not stated | Exact |
Showing 5 of 18 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Wolfram syndrome | 3463 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Wolfram syndrome | 3463 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.