Condition (MeSH)
Barth Syndrome
ClinicalTrials.gov lists 11 trials for Barth Syndrome or a narrower condition beneath it. Stealth BioTherapeutics Inc. has the most among industry and government lead sponsors, with 3.
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Trials per phase
11 of the 11 trials are tagged with Barth Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Barth Syndrome trials start? (8)
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 2/3
- Phase 3
- 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 |
| 2024 | 1 |
| 2018 | 1 |
| 2017 | 1 |
| 2012 | 2 |
| 2011 | 1 |
| 2010 | 1 |
| 2009 | 1 |
No forecast is made for this condition: it averages 0.4 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 Barth Syndrome trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 2/3 | 1 |
| Phase 3 | 1 |
| Phase 4 | 1 |
| Not applicable | 2 |
| Observational | 4 |
| Expanded access | 2 |
How many Barth Syndrome trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Active, not recruiting | 1 |
| Available | 1 |
| Completed | 4 |
| No longer available | 1 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Barth Syndrome 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 |
|---|---|
| Stealth BioTherapeutics Inc. | 3 |
| X4 Pharmaceuticals | 1 |
Which drugs are being tested for Barth 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 |
|---|---|
| Elamipretide | 3 |
| X4P-001 | 1 |
| Triheptanoin | 1 |
Which serious adverse events do Barth Syndrome trials report? (5)
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 |
|---|---|---|
| Abscess of Right Axilla | 1 | 1 |
| Costochondritis | 1 | 1 |
| Gastroenteritis | 1 | 1 |
| Mucositis | 1 | 1 |
| Pneumothorax | 1 | 1 |
Which publications cover Barth Syndrome? (5)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
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 |
|---|---|---|---|---|---|
| Barth syndrome | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Barth syndrome | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Barth syndrome | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Barth syndrome | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Barth syndrome | Prevalence at birth | 1-9 / 1 000 000 | United States | US | 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 |
|---|---|---|---|
| Barth syndrome | 111 | Exact | Orphadata Science knowledge files, Product 1 |
| Barth syndrome | 111 | 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.