Gene therapy zolgensma tested in kids with SMA who stopped other drugs

NCT ID NCT05386680

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

Kennedy disease muscle tissue disorder muscular atrophy spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINAL MUSCULAR ATROPHY

Contacts and locations

Locations

Boston Childrens Hospital

Boston, Massachusetts, 02215, United States
Child Hosp Of The Kings Daughters

Norfolk, Virginia, 23507, United States
Novartis Investigative Site

Parkville, Victoria, 3052, Australia
Novartis Investigative Site

Leuven, 3000, Belgium
Novartis Investigative Site

Montreal, Quebec, H4A 3J1, Canada
Novartis Investigative Site

Bron, 69677, France
Novartis Investigative Site

Toulouse, 31059, France
Novartis Investigative Site

Roma, RM, 00168, Italy
Novartis Investigative Site

Kurume, Fukuoka, 830-0011, Japan
Novartis Investigative Site

Shinjuku Ku, Tokyo, 162 8666, Japan
Novartis Investigative Site

Utrecht, 3584, Netherlands
Novartis Investigative Site

Barcelona, Catalonia, 08035, Spain
University of Wisconsin Madison Medical School

Madison, Wisconsin, 53792-7375, United States

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