ARGX-119 given by infusion to children with spinal muscular atrophy
Tests ARGX-119 given through a vein in children with spinal muscular atrophy.
Plain-language title generated from the registry record
Registry title: 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
Key facts
- Where
- Baltimore, Boston, Chicago, Columbia, Flower Mound, Hartford, Hillsborough, Houston, Iowa City, Kissimmee, Little Rock, New York, Norfolk, Philadelphia, San Diego, and Stanford, United StatesSee the 16 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 5 and 17 years old?
Source: NCT07287982, who can take part
Where in United States
- The Johns Hopkins HospitalBaltimore, United StatesNot yet recruiting
- Boston Children's HospitalBoston, United StatesTaking people
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- The Curators of the University of Missouri on behalf of University of Missouri Health CareColumbia, United StatesNot yet recruiting
- Neurology Rare Disease CenterFlower Mound, United StatesTaking people
- Connecticut Children's Medical CenterHartford, United StatesTaking people
- Rare Disease Research NC, LLCHillsborough, United StatesTaking people
- Texas Children's HospitalHouston, United StatesTaking people
- University of Iowa Stead Family Children's HospitalIowa City, United StatesTaking people
- Rare Disease Research FL LLCKissimmee, United StatesTaking people
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- Columbia University / Herbert Irving Comprehensive Cancer CenterNew York, United StatesTaking people
- Childrens Hospital of The Kings DaughtersNorfolk, United StatesNot yet recruiting
- Children's Hospital Philadelphia - NeurologyPhiladelphia, United StatesNot yet recruiting
- Rady Childrens HospitalSan Diego, United StatesNo longer taking part
- Stanford University Medical CenterStanford, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- argenx
- Trial code
- NCT07287982EU 2025-523496-32-00
- Record updated
- 8 October 2026
- Original description
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).Read full original descriptionShow less
A Phase 2 Double-Blinded, Randomized, Placebo-Controlled 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
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).
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: 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; 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; 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.