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NCT03709784 · ClinicalTrials.gov registry record
Spinraza in Adult Spinal Muscular Atrophy
A clinical trial of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, sponsored by Washington University School of Medicine.
- Active
- Registry status
- 148
- Enrollment target
- 11
- Study locations
NCT03709784: Active study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, sponsored by Washington University School of Medicine.
NCT03709784 is a study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3 that is active but no longer recruiting, run by Washington University School of Medicine. The registered enrollment target is 148 participants, above the 102-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (45% higher). The trial reports 11 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03709784, a study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, is active but no longer recruiting, sponsored by Washington University School of Medicine.
- ACTIVE NOT RECRUITING
- Registry status
- 148 participants
- Enrollment target
- 11
- Study locations
Study Summary
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.
Primary Outcome
Assess effectiveness of SPINRAZA® (nusinersen) treatment on mobility and ambulation in ambulatory adult SMA patients, comparing changes in total distance walked in in six minutes from baseline until end of treatment at 30 months..
Conditions Studied
Interventions
- DRUG Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
- OTHER One time survey
Study Locations (11)
Arizona
- Barrow Neurological Institute - Phoenix
District of Columbia
- Georgetown University - Washington D.C.
Maryland
- Johns Hopkins - Baltimore
Massachusetts
- Massachusetts General Hospital-Harvard University - Boston
Michigan
- Memorial Healthcare - Owosso
Missouri
- Washington University School of Medicine - St Louis
New York
- New York University School of Medicine - New York
Texas
- Houston Methodist Neurological Institute - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 148 participants |
| Start Date | 2018-08-16 |
| Est. Completion | 2025-01-01 |
What the registry record for NCT03709784 still lists
NCT03709784 is an observational study that tracks outcomes without assigning an intervention. The registered 148 participants enrollment target is mid-sized for trials with a published cap, above the 102-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (45% higher).
The record links to 3 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 2 interventions - of which Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care is the first listed.
NCT03709784 lists 11 locations in 11 states (Arizona, District of Columbia, Maryland).
Frequently Asked Questions
What is clinical trial NCT03709784 about?
NCT03709784 is a clinical study titled "Spinraza in Adult Spinal Muscular Atrophy". 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.
What is the current status of trial NCT03709784?
This trial is currently active not recruiting. The enrollment target is 148 participants. The study started on 2018-08-16. Estimated completion is 2025-01-01.
What conditions does trial NCT03709784 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II.
What interventions are being tested in trial NCT03709784?
The interventions under investigation include: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care (DRUG), One time survey (OTHER).
Who is sponsoring clinical trial NCT03709784?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03709784 being conducted?
This trial has 11 study locations across Arizona, District of Columbia, Maryland, Massachusetts, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03709784's enrollment target sits among peer trials
148 7th of 22 higher than 16 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
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