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A Study of ARGX-119 for Spinal Muscular Atrophy in Children

This study is testing a new treatment called ARGX-119 for children aged 5 to 17 years old who have Spinal Muscular Atrophy (SMA). The main goals are to find a safe and effective dose of ARGX-119, understand how it works in the body, and see how the immune system reacts to it. Participants will receive either ARGX-119 or a placebo (an inactive substance) intravenously (through a vein) for 24 weeks, in addition to their current SMA treatment. After this, all participants will receive ARGX-119 for up to 100 weeks. The study will measure side effects and changes in motor skills using a special score called the RHS total score. This study is currently recruiting participants.

Study design
This is a Phase 2 interventional study with a planned enrollment of 60 participants. It includes a double-blinded period where participants receive either ARGX-119 or a placebo, followed by an open-label period where all participants receive ARGX-119.
What's involved
You would participate in a double-blinded treatment period for 24 weeks, followed by an open-label active-treatment extension period for up to 100 weeks. This involves receiving intravenous infusions of ARGX-119 or placebo.
Compensation
Not stated in the trial record.
Follow-up
The study will track your safety for up to 124 weeks. Your motor skills will be assessed up to 24 weeks into the double-blinded treatment period.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07287982

A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

Recruiting
PHASE2Ages 5–17InterventionalTreatment
argenx
~60 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:ARGX-119 IVPlacebo IV

At a glance

Recruiting sites
14 of 23 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of AEs
Measured over Up to 124 weeks
+2 more outcomes measured

Conditions

Spinal Muscular Atrophy (SMA)

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Where it's being run

23 sites across 21 states
California2
Texas2
Arkansas1
Connecticut1
Florida1
Illinois1
Iowa1
Maryland1

Who to contact

Sabine Coppieters, MD
[email protected]
857-350-4834
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Incidence of AEsUp to 124 weeks

    Adverse Events

  • Incidence of SAEsUp to 124 weeks

    Serious Adverse Events

  • Change in RHS total score from baseline to week 24 of the double blinded treatment period (DBTP)Up to 24 weeks

    The RHS (Revised Hammersmith Scale) is a validated 36-item scale developed to evaluate the spectrum of gross motor function. Maximum total score; 69 (optimal motor function)