Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Spinal Muscular Atrophy (SMA) clinical trials
Every US clinical trial registered for Spinal Muscular Atrophy (SMA) — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
6 US clinical trials · 5 currently recruiting
The research picture
Spinal Muscular Atrophy (SMA) has 6 registered US clinical trials, 5 of them open to new participants right now — about 83% of the total.
- 5
- recruiting participants now
- 83%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 2
- top sponsor: Novartis Pharmaceuticals
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
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
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Spinal Muscular Atrophy (SMA) Trial Landscape
ClinicalTrials.gov lists 6 US studies indexed under Spinal Muscular Atrophy (SMA), and 5 of those are currently open to recruitment — roughly 83% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Spinal Muscular Atrophy (SMA) shows 1 late-stage studies (Phase 3 and Phase 4 combined) alongside 2 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Spinal Muscular Atrophy (SMA) is led by Novartis Pharmaceuticals with 2 indexed trials, alongside 4 other organizations in the top contributor list. The list on this page surfaces up to 6 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Spinal Muscular Atrophy (SMA)?
PlainTrial tracks 6 US clinical trials for Spinal Muscular Atrophy (SMA), of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Spinal Muscular Atrophy (SMA)?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.