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

NCT ID NCT05386680

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

27 people

The number who actually took part.

Started

Jan 2023

Finished

Nov 2024

Lead sponsor

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.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * SMA diagnosis * Aged 2 to \< 18 years * Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening * Must have symptoms of SMA as defined in the protocol Exclusion Criteria: * Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated * Clinically significant abnormalities in test results during screening * Contraindications for lumbar puncture procedure * At Baseline, participants are excluded if they received: * nusinersen (Spinraza®) or * risdiplam (Evrysdi®) within a defined timeframe * Vaccinations 2 weeks prior to administration of OAV101 * Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned. * Presence of an infection or febrile illness up to 30 days prior to administration of OAV101 * Requiring invasive ventilation

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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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