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New hope for kids with SMA: experimental drug ARGX-119 enters Mid-Stage trial

NCT ID NCT07287982

Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 28, 2026 · Updated 9 times

Summary

This study tests a new biologic drug called ARGX-119 in children aged 5 to 17 with spinal muscular atrophy (SMA). About 60 participants will receive either ARGX-119 or a placebo by IV infusion for 24 weeks, alongside their usual SMA therapy. The goal is to find the best dose and see if it safely improves muscle function and walking ability.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ARGX-119 (a biologic drug given by IV infusion)
What this could lead to
If successful, ARGX-119 could become an additional treatment option to improve motor function in children with spinal muscular atrophy.
What could go wrong
This is a phase 2 trial with only 60 participants, so results may not apply to all. The drug is still experimental, and side effects or lack of benefit are possible.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

May 2029

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

5 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: * Is aged ≥5 to \<18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF, and can comply with protocol * requirements. * Has documented historical genetic diagnosis of 5q-SMA. * Currently receiving a stable SMA treatment regimen (nusinersen or risdiplam) and/or have a history of onasemnogene abeparvovec treatment * Must be able to walk at least 50 meters without walking aids in the 6MWT at screening Exclusion Criteria: * Known medical condition that would interfere with an accurate assessment of SMA, confound the results of the study, or put the participant at undue risk, as assessed by the investigator * Recent major surgery, except spinal fusion, within 3 months of screening or intends to have major surgery during the study * Current or previous administration of antimyostatin therapies in the past 6 months * Severe scoliosis (defined as curvature \>40°) and/or contractures at screening. o History of spinal fusion within 6 months before screening or planned during the study * Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for daytime treatment while awake. Ventilation used overnight or during daytime naps is acceptable.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

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

Spinal muscular atrophy (SMA)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    22 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Addenbrooke's Hospital

    RECRUITING

    Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

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

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Birmingham Heartlands Hospital

    RECRUITING

    Birmingham, West Midlands, B9 5SS, United Kingdom

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Philadelphia - Neurology

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital of The Kings Daughters

    NOT_YET_RECRUITING

    Norfolk, Virginia, 23507, United States

  • Columbia University Herbert Irving Comprehensive Cancer Center

    NOT_YET_RECRUITING

    New York, New York, 10032, United States

  • Connecticut Children's Medical Center

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • Great Ormond Street Hospital for Children

    NOT_YET_RECRUITING

    London, Greater London, WC1N 3JH, United Kingdom

  • Leeds General Infirmary

    RECRUITING

    Leeds, West Yorkshire, LS1 3EX, United Kingdom

  • MDUK Oxford Neuromuscular Centre

    NOT_YET_RECRUITING

    Oxford, Oxfordshire, OX3 9DU, United Kingdom

  • Neurology Rare Disease Center

    RECRUITING

    Flower Mound, Texas, 75028, United States

  • Rady Childrens Hospital

    WITHDRAWN

    San Diego, California, 92123, United States

  • Rare Disease Research FL LLC

    RECRUITING

    Kissimmee, Florida, 34746, United States

  • Rare Disease Research NC, LLC

    RECRUITING

    Hillsborough, North Carolina, 27278, United States

  • Royal Hospital for Children

    RECRUITING

    Glasgow, Strathclyde, G51 4TF, United Kingdom

  • Sheffield Children's Hospital

    RECRUITING

    Sheffield, South Yorkshire, S10 2TH, United Kingdom

  • Stanford University Medical Center

    RECRUITING

    Stanford, California, 94305, United States

  • Texas Children's Hospital

    NOT_YET_RECRUITING

    Houston, Texas, 77030, United States

  • The Curators of the University of Missouri on behalf of University of Missouri Health Care

    NOT_YET_RECRUITING

    Columbia, Missouri, 65212, United States

  • The Johns Hopkins Hospital

    NOT_YET_RECRUITING

    Baltimore, Maryland, 21205, United States

  • University of Iowa Stead Family Children's Hospital

    RECRUITING

    Iowa City, Iowa, 52242, United States

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