Can gene therapy zolgensma safely improve motor skills in SMA?
NCT ID NCT07767890
First seen Aug 17, 2026 · Last updated Sep 18, 2026 · Updated 3 times
Summary
This study follows 80 people with spinal muscular atrophy (SMA) in Japan who receive Zolgensma (onasemnogene abeparvovec) as an intrathecal injection. The goal is to track safety issues, such as liver problems or blood clotting abnormalities, and to measure changes in motor function using a standard SMA assessment scale. Since it's an observational study, patients receive standard care, and researchers simply collect data from their doctors. The findings could help clarify how well this gene therapy works in real-world clinical practice.
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 (Zolgensma) given as an intrathecal injection
- What this could lead to
- If successful, this study could provide real-world safety and effectiveness data for Zolgensma in Japanese SMA patients, potentially guiding treatment decisions and improving patient outcomes.
- What could go wrong
- This is an observational study, so it cannot prove cause and effect. The small size (80 patients) and single-country setting may limit how well the findings apply to broader populations.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2026
An estimate. Start dates often move.
- Expected to finish
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Mar 2031
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.
- Ages
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2 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion criteria: • All patients treated with onasemnogene abeparvovec. Exclusion criteria: • Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
- Can a patient registry unlock the secrets of spinal muscular atrophy?