Condition (MeSH)
Myopathies, Structural, Congenital
ClinicalTrials.gov lists 32 trials for Myopathies, Structural, Congenital or a narrower condition beneath it. Astellas Gene Therapies has the most among industry and government lead sponsors, with 5.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
23 of the 32 trials are tagged with Myopathies, Structural, Congenital itself; the rest are tagged with a narrower condition beneath it.
When did Myopathies, Structural, Congenital 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 1/2
- Phase 2
- Phase 2/3
- 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) | 4 |
| 2025 | 6 |
| 2024 | 4 |
| 2022 | 1 |
| 2021 | 1 |
| 2020 | 3 |
| 2018 | 2 |
| 2017 | 2 |
| 2016 | 1 |
| 2015 | 1 |
Showing 10 of 13 earlier years.
No forecast is made for this condition: it averages 3.0 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 Myopathies, Structural, Congenital trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1/2 | 4 |
| Phase 2 | 1 |
| Phase 2/3 | 1 |
| Not applicable | 8 |
| Observational | 18 |
How many Myopathies, Structural, Congenital trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 5 |
| Recruiting | 11 |
| Active, not recruiting | 2 |
| Completed | 9 |
| Terminated | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Myopathies, Structural, Congenital trials? (3)
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 |
|---|---|
| Astellas Gene Therapies | 5 |
| Dynacure | 3 |
| Oculox Technologies SA | 1 |
Which drugs are being tested for Myopathies, Structural, Congenital? (5)
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 |
|---|---|
| Tyrosine | 1 |
| PREDNISOLONE | 1 |
| Albuterol | 1 |
| Sirolimus | 1 |
| METHYLPREDNISOLONE | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| DYN101 | 2 |
| ApoTamox 10mg | 1 |
| QOL assessment, Neuropsychological | 1 |
| Salbutamol Only Product in Oral Dose Form | 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 Myopathies, Structural, Congenital 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 |
|---|---|---|
| Ascites | 3 | 1 |
| Hyperbilirubinaemia | 3 | 1 |
| Viral upper respiratory tract infection | 3 | 1 |
| Cholesteatoma | 2 | 2 |
| Dehydration | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Myopathies, Structural, Congenital? (44)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial. | The Lancet. Neurology | 2023 | Yes |
| Whole genome sequencing reveals biallelic PLA2G6 mutations in siblings with cerebellar atrophy and cap myopathy. | Clinical genetics | 2021 | Yes |
| Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis. | Archives of disease in childhood | 2020 | Yes |
| X-linked myotubular myopathy: A prospective international natural history study. | Neurology | 2019 | Yes |
| The Use of Muscle Ultrasound in the Diagnosis and Differential Diagnosis of Congenital Disorders of Muscle in the Age of Next Generation Genetics. | Seminars in pediatric neurology | 2019 | Yes |
Showing 5 of 44 publications.
Sub-conditions in MeSH (2)
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.