ARGX-119 IV for ambulant children aged 5 to under 18 with spinal muscular atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind Phase 2 trial that begins with a 24-week period in which ambulant pediatric participants receive either ARGX-119 IV or placebo IV alongside their stable disease-modifying therapy. The study will identify an age-appropriate dose and collect safety, tolerability, pharmacokinetics, immunogenicity, and preliminary efficacy data focused on muscle function and fatigability. Participants who complete the blinded period may enter an open-label extension of up to about 100 weeks during which all participants receive ARGX-119 IV. The investigational approach targets MuSK to potentially stabilize neuromuscular junction function as a complement to existing SMA therapies.

Why it matters

Who can join: the full rules

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.

Where it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Stanford University Medical Center
  Stanford, California, United States Recruiting
• Connecticut Children's Medical Center
  Hartford, Connecticut, United States Recruiting
• Rare Disease Research FL LLC
  Kissimmee, Florida, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• University of Iowa Stead Family Children's Hospital
  Iowa City, Iowa, United States Recruiting

Study contacts

• Study coordinator: Sabine Coppieters, MD
• Email: Clinicaltrials@argenx.com
• Phone: 857-350-4834

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Wearable monitoring of motor development in babies and young children with spinal muscular atrophy detected at birth

  This project will try wearing small motion sensors at home to see if they can track how babies and young children diagnosed with spinal muscular atrophy at birth move and develop, compared with healthy children.

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• HABIT-ILE plus Functional Strength Training for Children with SMA

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• Long-read genetic analysis for Spinal Muscular Atrophy (LOREASI)

  This project tries a long-read DNA technique on adults with known SMN1 copy-number patterns to find hidden '2+0' carrier genotypes that standard tests miss.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.