Gene therapy zolgensma tested in kids with SMA who can sit but not stand
NCT ID NCT03381729
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety and how well children tolerated different doses.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Onasemnogene abeparvovec-xioi (Zolgensma), a gene therapy delivered via spinal injection
- What this could lead to
- If successful, this could provide a treatment option for children with spinal muscular atrophy who can sit but not yet stand or walk, potentially improving motor function.
- What could go wrong
- This is an early phase 1 trial with only 32 participants, so results are preliminary. The trial was terminated, which may limit data. Gene therapy carries risks like immune reactions or liver issues.
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
Chicago, Illinois, 60611, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Johns Hopkins
Baltimore, Maryland, 21287, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Stanford University
Stanford, California, 94305, United States
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UCLA
Los Angeles, California, 90095, United States
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UT Southwestern
Dallas, Texas, 75390, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Washington University
St Louis, Missouri, 63130, United States
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