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Gene therapy shows promise for kids with SMA type 2

NCT ID NCT05089656

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a one-time gene therapy called OAV101 in 126 children aged 2 to 18 with type 2 spinal muscular atrophy (SMA) who had never received other SMA treatments. The therapy was given as a spinal injection and compared to a sham procedure. The goal was to see if it could improve motor skills like sitting and moving. While not a cure, it aims to help control the disease and improve daily function.

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

126 people

The number who actually took part.

Started

Feb 2022

Finished

Apr 2025

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.

Key Inclusion criteria: * Diagnostic confirmation during screening period of 5q SMA * The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)). * Onset of clinical signs and symptoms at ≥ 6 months of age * A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility * Able to sit independently at screening, but has never had the ability to walk independently. Key Exclusion criteria: * Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to \> 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab. * Infectious process (e.g., viral, bacterial) or febrile illness within 30 days prior to OAV101 treatment or sham procedure * Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), \> upper limit of normal (ULN). * Requiring invasive ventilation, awake noninvasive ventilation for \> 6 hours during a 24-hour period, noninvasive ventilation for \> 12 hours during a 24-hour period or requiring tracheostomy * Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle \> 40 in a sitting position * Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks * Clinically significant sensory abnormalities in the neurological examination at Screening

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Conditions

The condition(s) this trial relates to.

muscle tissue disorder muscular atrophy Spinal Muscular Atrophies of Childhood spinal muscular atrophy spinal muscular atrophy, type II

As listed by the trial registrant

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

Type 2 spinal muscular atrophy

Contacts and locations

Locations

  • AIIMS, Ansari Nagar

    New Delhi, New Delhi, 110029, India

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Child Hosp Of The Kings Daughters

    Norfolk, Virginia, 23507, United States

  • Children's Specialty Group/CHKD

    Norfolk, Virginia, 23507, United States

  • Clinic for Special Children

    Strasburg, Pennsylvania, 17579, United States

  • Connecticut Children's Medical Center

    Farmington, Connecticut, 06032, United States

  • Hospital Civil De Guadalajara Fray Antonio Alcalde

    Guadalajara, Jalisco, 44280, Mexico

  • Kaohsiung Medical University Hospital

    Kaohsiung City, 80756, Taiwan

  • National Children's Hospital

    Hanoi, 100000, Vietnam

  • Novartis Investigative Site

    Curitiba, Paraná, 81520-060, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    Beijing, Beijing Municipality, 100034, China

  • Novartis Investigative Site

    Chongqing, Chongqing Municipality, 400010, China

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510623, China

  • Novartis Investigative Site

    Shenzhen, Guangdong, 518034, China

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041, China

  • Novartis Investigative Site

    Hangzhou, Zhejiang, 310052, China

  • Novartis Investigative Site

    Beijing, 100069, China

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Copenhagen, 2100 O, Denmark

  • Novartis Investigative Site

    New Delhi, National Capital Territory of Delhi, 110029, India

  • Novartis Investigative Site

    New Delhi, National Capital Territory of Delhi, 110060, India

  • Novartis Investigative Site

    Kolkata, West Bengal, 700094, India

  • Novartis Investigative Site

    Hyderabad, 500034, India

  • Novartis Investigative Site

    Mumbai, 400016, India

  • Novartis Investigative Site

    Kuala Lumpur, 50300, Malaysia

  • Novartis Investigative Site

    Kuala Lumpur, 59100, Malaysia

  • Novartis Investigative Site

    Guadalajara, Jalisco, 44280, Mexico

  • Novartis Investigative Site

    Mexico City, Mexico City, 06720, Mexico

  • Novartis Investigative Site

    Riyadh, 11211, Saudi Arabia

  • Novartis Investigative Site

    Singapore, 119074, Singapore

  • Novartis Investigative Site

    Cape Town, 7925, South Africa

  • Novartis Investigative Site

    Kaohsiung City, 80756, Taiwan

  • Novartis Investigative Site

    Bangkok, 10700, Thailand

  • Novartis Investigative Site

    Hanoi, 100000, Vietnam

  • P.D. Hinduja National Hospital & MRC

    Mumbai, 400016, India

  • Paediatric Neurology

    Copenhagen, 2100 O, Denmark

  • Peking University First Hospital

    Beijing, 100034, China

  • Red Cross War Memorial Childrens Hospital

    Cape Town, 7925, South Africa

  • Sir Ganga Ram Hospital

    New Delhi, National Capital Territory of Delhi, 110 060, India

  • Siriraj Hospital

    Bangkok, 10700, Thailand

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

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