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Zolgensma's long-term effects under the microscope in new 5-year study

NCT ID NCT05335876

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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 10, 2026 · Updated 2 times

Summary

This study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective the treatment remains over time.

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)
What this could lead to
If successful, this study could confirm that a one-time dose of Zolgensma provides lasting benefits and is safe over many years for people with spinal muscular atrophy.
What could go wrong
This is a follow-up study, not a new treatment test. It may reveal unknown long-term side effects, and results depend on how well patients did in earlier trials.

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

20 people

The number who actually took part.

Started

Dec 2022

Expected to finish

Apr 2031

An estimate. End dates often move.

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

0 to 100 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: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. Exclusion Criteria: There are no exclusion criteria for this study.

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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 (SMA)

Contacts and locations

Locations

  • Child Hosp Of The Kings Daughters

    Norfolk, Virginia, 23507, United States

  • Novartis Investigative Site

    Sydney, New South Wales, 2031, Australia

  • Novartis Investigative Site

    Leuven, 3000, Belgium

  • Novartis Investigative Site

    Curitiba, Paraná, 81520-060, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    Montreal, Quebec, H4A 3J1, Canada

  • Novartis Investigative Site

    Chongqing, Chongqing Municipality, 400010, China

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510623, China

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041, China

  • Novartis Investigative Site

    Beijing, 100034, China

  • Novartis Investigative Site

    Beijing, 100069, China

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Copenhagen, 2100 O, Denmark

  • Novartis Investigative Site

    Bron, 69677, France

  • Novartis Investigative Site

    Garches, 92380, France

  • Novartis Investigative Site

    Strasbourg, 67000, 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, 1628666, Japan

  • Novartis Investigative Site

    Kuala Lumpur, 50300, Malaysia

  • Novartis Investigative Site

    Kuala Lumpur, 59100, Malaysia

  • Novartis Investigative Site

    Utrecht, 3584 CX, Netherlands

  • Novartis Investigative Site

    Riyadh, 11211, Saudi Arabia

  • Novartis Investigative Site

    Singapore, 119074, Singapore

  • Novartis Investigative Site

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    Kaohsiung City, 80756, Taiwan

  • Novartis Investigative Site

    Taipei, 10002, Taiwan

  • Novartis Investigative Site

    Bangkok, 10700, Thailand

  • Novartis Investigative Site

    London, WC1N 3JH, United Kingdom

  • Novartis Investigative Site

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • Novartis Investigative Site

    Hanoi, 100000, Vietnam

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