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 now · Phase 2 · Has a placebo group

Conditions studied: Spinal Muscular Atrophy (SMA)

In brief

This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).

Key facts

Study ID

NCT07287982

Run by

argenx

People needed

60

Starts

2025-12-19

Expected to finish

2029-05-28

Last updated by the study team

2026-07-21

Who can join

Age: 5 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 is running

• Rare Disease Research NC, LLC — Hillsborough, North Carolina, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Connecticut Children's Medical Center — Hartford, Connecticut, United States (enrolling)
• Neurology Rare Disease Center — Flower Mound, Texas, United States (enrolling)
• Rare Disease Research FL LLC — Kissimmee, Florida, United States (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• The Curators of the University of Missouri on behalf of University of Missouri Health Care — Columbia, Missouri, United States
• Columbia University Herbert Irving Comprehensive Cancer Center — New York, New York, United States
• Children's Hospital Philadelphia - Neurology — Philadelphia, Pennsylvania, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• Texas Children's Hospital — Houston, Texas, United States
• Childrens Hospital of The Kings Daughters — Norfolk, Virginia, United States
• Rady Childrens Hospital — San Diego, California, United States
• Stanford University Medical Center — Stanford, California, United States
• University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States
• The Johns Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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