Last reviewed · How we verify
NCT03709784: SAS
Spinraza in Adult Spinal Muscular Atrophy
trial testing Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care in Spinal Muscular Atrophy in 148 participants. Participants enrolled and being followed up; not accepting new ones.
31 December 2024
Quick facts
| Lead sponsor | Washington University School of Medicine |
|---|---|
| Status | Active, enrolled |
| Study type | OBSERVATIONAL |
| Enrollment | 148 |
| Start date | 16 August 2018 |
| Primary completion | 31 December 2024 |
| Estimated completion | 1 January 2025 |
| Sites | 11 locations across Canada, United States |
Drugs / interventions tested
- Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
- One time survey
Conditions studied
- Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →
- Spinal Muscular Atrophy Type II — all drugs for Spinal Muscular Atrophy Type II →
- Spinal Muscular Atrophy Type 3 — all drugs for Spinal Muscular Atrophy Type 3 →
Sponsor
Washington University School of Medicine
Who can join
Adults 18 to 70, any sex, with Spinal Muscular Atrophy or Spinal Muscular Atrophy Type II. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Drug treatment for spinal muscular atrophy types II and III.
Wadman RI, van der Pol WL, Bosboom WM, Asselman FL, et al · · 2020 · cited 32× · PMID 32006461 · DOI 10.1002/14651858.cd006282.pub5 -
Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical-Experimental, and Real-World Evidence.
Crisafulli S, Boccanegra B, Vitturi G, Trifirò G, et al · · 2023 · cited 11× · PMID 37891814 · DOI 10.3390/brainsci13101446
Verify or expand the search:
- PubMed search for NCT03709784
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Spinal Muscular Atrophy
Currently open trials in the same condition.
- NCT07478172 — Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease · NA · recruiting
- NCT06955897 — Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA · recruiting
- NCT06396325 — A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With · NA · recruiting
- NCT06288230 — An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy · Phase 1, PHASE2 · recruiting
- NCT06321965 — Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy · NA · recruiting
Other Washington University School of Medicine trials
Trials by the same sponsor.
- NCT05521997 — Glutaminase Inhibition and Chemoradiation in Advanced Cervical Cancer · Phase 2 · not yet recruiting
- NCT07101666 — Total Neoadjuvant Therapy With Short Course Radiation Therapy in Gastric Cancer · Phase 2 · not yet recruiting
- NCT07200089 — Recombinant Human IL-7 (NT-I7) in Relapsed/Refractory Multiple Myeloma Following BCMA CAR-T Therapy (Cilta-cel) · Phase 1 · not yet recruiting
- NCT07313592 — Whole Genome Sequencing (ChromoSeq®) for Acute Lymphoblastic Leukemia (ALL) Patients · not yet recruiting
- NCT07419464 — 5-Fluorouracil Response and Optimization STudy (The FROST Trial) · Phase 2 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT03709784 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Washington University School of Medicine
- Last refreshed: 20 November 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03709784.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing