Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Spinal Muscular Atrophy (SMA): early-phase trial registry
5 of 6 indexed Spinal Muscular Atrophy (SMA) trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,866 of 28,707 conditions by registered trial count.
6 US clinical trials · 5 currently recruiting · #2,866 of 28,707 conditions by registered trial count
Spinal Muscular Atrophy (SMA): 6 US clinical trials tracked, 5 recruiting.
Spinal Muscular Atrophy (SMA) is the subject of 6 registered US clinical trials on ClinicalTrials.gov, 5 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is Novartis Pharmaceuticals, running 2 of these trials.
Enrollment posture brief
Registry condition spinal-muscular-atrophy-sma
OPEN 5 | RECR 83% | LATE 1 | EARLY 2 | RANK #2866 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Alcoholic Hepatitis (83% recruiting · 6 trials)
Spinal Muscular Atrophy (SMA) lists 5 open slots (83% of 6 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Spinal Muscular Atrophy (SMA) on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
-
Spinal Muscular Atrophy (SMA) is recruiting above its size-band peers.
5 of 6 indexed trials (83.3%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
-
One sponsor concentrates Spinal Muscular Atrophy (SMA) research.
Novartis Pharmaceuticals accounts for 2 of 6 trials (33.3%). 5 distinct sponsors appear on at least one study for this condition label.
-
Typical Spinal Muscular Atrophy (SMA) enrolment sits around 200 participants.
Median target enrolment is 200 among the 6 trials that report a genuine target (sentinel values excluded). 2 of those (33.3%) plan for fewer than 100 participants.
-
Spinal Muscular Atrophy (SMA) splits between interventional and observational designs.
4 interventional and 2 observational studies make up this index (66.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
6 total, page 1 of 1
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Novartis Pharmaceuticals
NCT04174157
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Medstar Health Research Institute
NCT07223489
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Novartis Pharmaceuticals
NCT05335876
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
argenx
NCT07287982
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
Teachers College, Columbia University
NCT07488338
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Hoffmann-La Roche
NCT05115110
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 2 |
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Spinal Muscular Atrophy (SMA) records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Spinal Muscular Atrophy (SMA) trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Spinal Muscular Atrophy (SMA) page
6 registered trials, 5 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 5 Spinal Muscular Atrophy (SMA) trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Novartis Pharmaceuticals sponsors the most Spinal Muscular Atrophy (SMA) trials on record, review their full trial history before evaluating a specific study. See Novartis Pharmaceuticals's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Spinal Muscular Atrophy (SMA) condition card totals 6 registered trials; most-linked intervention RO7204239; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.