SPINRAZA study tracks movement in adults with spinal muscular atrophy
NCT ID NCT04159987
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINRAZA works in adults with this condition.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- SPINRAZA (nusinersen)
- What this could lead to
- If successful, this study could confirm that SPINRAZA helps maintain or improve motor function in adults with SMA type II, supporting its continued use.
- What could go wrong
- This is a small, open-label study with only 20 participants and no placebo group, so results may not apply broadly. It tracks function but does not test a new treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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APHP
Paris, Île-de-France Region, 94000, France
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CHRU de Lille
Lille, Hauts-de-France, 59000, France
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CHU de Lyon
Lyon, Auvergne-Rhône-Alpes, 69677, France
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CHU de Montpellier
Montpellier, Occitanie, 34090, France
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CHU de Nice
Nice, Provence-Alpes-Côte d'Azur Region, 06000, France
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CHU de Toulouse
Toulouse, Occitanie, 31059, France
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Hopital de la Timone - APHM
Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can gene therapy help babies with SMA reach milestones? a Real-World review
- School transitions put to the test for children with a rare muscle disease
- Horseback therapy may boost movement and breathing in kids with Muscle-Weakening disease
- Newborn screening study aims to catch rare diseases at birth
- Gene therapy trial aims to halt Muscle-Wasting disease
- Higher dose of spinraza tested in SMA patients who already tried risdiplam