Condition (MeSH)
Spinal Muscular Atrophies of Childhood
ClinicalTrials.gov lists 60 trials for Spinal Muscular Atrophies of Childhood or a narrower condition beneath it.
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Trials per phase
60 of the 60 trials are tagged with Spinal Muscular Atrophies of Childhood itself; the rest are tagged with a narrower condition beneath it.
When did Spinal Muscular Atrophies of Childhood trials start? (17)
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 3
- Phase 4
- Not applicable
- Observational
Top of scale: 7
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 | 4 |
| 2024 | 5 |
| 2023 | 7 |
| 2022 | 7 |
| 2021 | 1 |
| 2020 | 5 |
| 2019 | 5 |
| 2018 | 3 |
| 2017 | 3 |
Showing 10 of 17 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 5 trials starting in 2026 and about 5 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 13 trials still running at the 2026-10-09 snapshot; registered median of 21 months across 32 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Spinal Muscular Atrophies of Childhood trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 9 |
| Phase 2 | 7 |
| Phase 3 | 5 |
| Phase 4 | 1 |
| Not applicable | 17 |
| Observational | 19 |
How many Spinal Muscular Atrophies of Childhood 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 | 2 |
| Recruiting | 9 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 7 |
| Completed | 28 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Spinal Muscular Atrophies of Childhood trials? (6)
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 |
|---|---|
| Novartis Gene Therapies | 4 |
| Scholar Rock, Inc. | 4 |
| MarsiBionics | 3 |
| GeneCradle Inc | 2 |
| Guangzhou Jiayin Biotech Ltd | 2 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Spinal Muscular Atrophies of Childhood? (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 |
|---|---|
| Apitegromab | 4 |
| Pyridostigmine | 2 |
Interventions as named in the registry (13)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Onasemnogene Abeparvovec-xioi | 3 |
| EXG001-307 injection | 2 |
| 4-Phenylbutyrate | 2 |
| Vital signs | 2 |
| Paracetamol 120Mg/5mL Oral Suspension | 1 |
Showing 5 of 13 interventions.
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 Spinal Muscular Atrophies of Childhood 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 |
|---|---|---|
| Pneumonia | 35 | 2 |
| Respiratory, thoracic and mediastinal disorders - Other, specify | 13 | 1 |
| Atelectasis | 12 | 4 |
| Vomiting | 10 | 2 |
| Lower respiratory tract infection | 8 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Spinal Muscular Atrophies of Childhood? (95)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study. | Neurology | 2025 | Yes |
| Risdiplam in Presymptomatic Spinal Muscular Atrophy. | The New England journal of medicine | 2025 | Yes |
| Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial. | The Lancet. Neurology | 2025 | Yes |
| The effect of thoracolumbosacral orthosis on scoliosis progression and chest deformity in children with type 1 spinal muscular atrophy: A randomized controlled trial. | PloS one | 2025 | Yes |
| Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. | The Journal of clinical investigation | 2025 | Yes |
Showing 5 of 95 publications.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence, rare-disease identity, and sub-conditions in mesh.