ARGX-119 IV for ambulant children aged 5 to under 18 with spinal muscular atrophy
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 trial will test whether IV ARGX-119, given with standard SMA therapies, is safe and can reduce muscle weakness and tiredness in children aged 5 to under 18.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 5 Years to 17 Years |
| Sex | All |
| Sponsor | argenx Industry-sponsored |
| Locations | 17 sites (Little Rock, Arkansas and 16 other locations) |
| Trial ID | NCT07287982 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are ambulant children aged 5 to under 18 with a confirmed 5q-SMA diagnosis who can walk at least 50 meters and are on a stable SMA treatment regimen (nusinersen, risdiplam, or prior onasemnogene abeparvovec).
Not a fit: Patients who are non-ambulatory, have severe scoliosis, recent major surgery, recent antimyostatin treatment, or other conditions that confound SMA assessment are less likely to benefit or be eligible for this protocol.
Why it matters
Potential benefit: If successful, ARGX-119 could improve neuromuscular junction function and reduce muscle weakness and fatigability, leading to better walking ability and quality of life for ambulant children with SMA.
How similar studies have performed: This MuSK-activation approach is relatively novel in SMA; other NMJ-targeting strategies and antimyostatin programs have shown mixed or preliminary results, so clinical proof of concept in SMA is limited.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Little Rock, Arkansas and 16 other locations
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
- Rady Childrens Hospital — San Diego, California, United States (Not_yet_recruiting)
- Stanford University Medical Center — Stanford, California, United States (Not_yet_recruiting)
- Connecticut Children's Medical Center — Hartford, Connecticut, United States (Not_yet_recruiting)
- Rare Disease Research FL LLC — Kissimmee, Florida, United States (Recruiting)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Not_yet_recruiting)
- University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States (Not_yet_recruiting)
- The Johns Hopkins Hospital — Baltimore, Maryland, United States (Not_yet_recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
- The Curators of the University of Missouri on behalf of University of Missouri Health Care — Columbia, Missouri, United States (Not_yet_recruiting)
- Columbia University Herbert Irving Comprehensive Cancer Center — New York, New York, United States (Not_yet_recruiting)
- Rare Disease Research NC, LLC — Hillsborough, North Carolina, United States (Recruiting)
- Children's Hospital Philadelphia - Neurology — Philadelphia, Pennsylvania, United States (Not_yet_recruiting)
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Not_yet_recruiting)
- Neurology Rare Disease Center — Flower Mound, Texas, United States (Recruiting)
- Texas Children's Hospital — Houston, Texas, United States (Not_yet_recruiting)
- Childrens Hospital of The Kings Daughters — Norfolk, Virginia, United States (Not_yet_recruiting)
Study contacts
- Study coordinator: Sabine Coppieters, MD
- Email: Clinicaltrials@argenx.com
- Phone: 857-350-4834
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.