Gene therapy zolgensma tested in kids with SMA who stopped other drugs
NCT ID NCT05386680
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a one-time gene therapy called OAV101 (Zolgensma) in 27 children aged 2 to 18 with spinal muscular atrophy (SMA) who had previously stopped taking other SMA medications (Spinraza or Evrysdi). The goal was to see if the treatment is safe and can help maintain or improve muscle function. The therapy is given as a single injection into the spinal fluid.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
OAV101 (Zolgensma), a gene therapy given as a one-time spinal injection
What this could lead to
If successful, this could offer a one-time gene therapy option for children with SMA who have stopped other treatments, potentially stabilizing or improving motor function.
What could go wrong
This is a small, single-arm study (27 participants) with no comparison group, so results may not be generalizable. Risks include liver toxicity, low platelets, and other serious side effects.
Disclaimer
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the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Boston Childrens Hospital
Boston, Massachusetts, 02215, United States
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Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507, United States
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Novartis Investigative Site
Parkville, Victoria, 3052, Australia
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Novartis Investigative Site
Leuven, 3000, Belgium
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Novartis Investigative Site
Montreal, Quebec, H4A 3J1, Canada
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Novartis Investigative Site
Bron, 69677, France
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Novartis Investigative Site
Toulouse, 31059, France
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Novartis Investigative Site
Roma, RM, 00168, Italy
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Novartis Investigative Site
Kurume, Fukuoka, 830-0011, Japan
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Novartis Investigative Site
Shinjuku Ku, Tokyo, 162 8666, Japan
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Novartis Investigative Site
Utrecht, 3584, Netherlands
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Novartis Investigative Site
Barcelona, Catalonia, 08035, Spain
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University of Wisconsin Madison Medical School
Madison, Wisconsin, 53792-7375, United States