Condition (MeSH)
Sarcoglycanopathies
ClinicalTrials.gov lists 7 trials for Sarcoglycanopathies or a narrower condition beneath it. Sarepta Therapeutics, Inc. has the most among industry and government lead sponsors, with 2.
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Trials per phase
7 of the 7 trials are tagged with Sarcoglycanopathies itself; the rest are tagged with a narrower condition beneath it.
When did Sarcoglycanopathies trials start? (6)
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
- 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 |
|---|---|
| 2024 | 1 |
| 2023 | 1 |
| 2021 | 2 |
| 2018 | 1 |
| 2015 | 1 |
| 2008 | 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: none of the 7 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Sarcoglycanopathies trials in? (3)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Observational | 5 |
How many Sarcoglycanopathies trials are recruiting? (4)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 1 |
| Active, not recruiting | 1 |
| Completed | 3 |
| Unknown status | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Sarcoglycanopathies 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 |
|---|---|
| Sarepta Therapeutics, Inc. | 2 |
| Ankara Etlik City Hospital | 1 |
Which serious adverse events do Sarcoglycanopathies trials report? (3)
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 |
|---|---|---|
| Rhabdomyolysis | 2 | 1 |
| Femur fracture | 1 | 1 |
| Myoglobinuria | 1 | 1 |
Which publications cover Sarcoglycanopathies? (5)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. | Nature medicine | 2024 | Yes |
| Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study. | BMC neurology | 2024 | Yes |
| Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. | Human gene therapy | 2019 | Yes |
| Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. | Molecular therapy : the journal of the American Society of Gene Therapy | 2017 | Yes |
| Natural History of Cardiac and Respiratory Involvement, Prognosis and Predictive Factors for Long-Term Survival in Adult Patients with Limb Girdle Muscular Dystrophies Type 2C and 2D. | PloS one | 2016 | Yes |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Qualitative or quantitative defects of sarcoglycan | 207052 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Qualitative or quantitative defects of sarcoglycan | 207052 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and sub-conditions in mesh.
Not computed for conditions with fewer than 10 trials: drugs tested.