Spinal injection drug shows promise in stopping SMA before symptoms start
NCT ID NCT02386553
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a drug called nusinersen (Spinraza) in 25 infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug is given through a spinal injection to help delay or prevent the need for breathing support or early death. The goal was to see if early treatment can keep these babies healthier longer.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Nusinersen (Spinraza), a drug given via spinal injection
- What this could lead to
- If it works, this could help infants with SMA grow up without needing breathing support or dying early.
- What could go wrong
- This is a small, early-phase study with only 25 infants, so results may not apply to all. The drug requires repeated spinal injections, which carry risks like infection or headache.
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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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Columbia University
New York, New York, 10032, United States
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David Geffen School of Medicine
Los Angeles, California, 90095, United States
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Hacettepe University Medical Faculty
Ankara, 6230, Turkey (Türkiye)
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Hamad General Hospital
Doha, 3050, Qatar
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Nemours Children's Hospital, Orlando
Orlando, Florida, 32827, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165, Italy
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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University of California Davis Health System
Sacramento, California, 95817, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Yeditepe University Medical School Hospital
Istanbul, 31755, Turkey (Türkiye)
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