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Clinical Trials in Australia / NCT07287982
Recruiting Phase 2

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

NCT07287982 · tracked via the Priya Life Science Australia tracker
Sponsor
argenx
Phase
Phase 2
Started
2025-12-19
Last updated
2026-10-08

Condition(s) studied

Spinal Muscular Atrophy (SMA)

Investigational drug(s) / intervention(s)

ARGX-119 IV →Placebo IV

ARGX-119 IV: Intravenous infusion of ARGX-119

Placebo IV: Intravenous infusion of placebo

Study summary

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).

Eligibility

Sex
ALL
Min age
5 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Incidence of AEs — Up to 124 weeks
    Adverse Events
  • Incidence of SAEs — Up 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)

Trial sites (28)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas Recruiting
Rady Childrens Hospital San Diego California Withdrawn
Stanford University Medical Center Stanford California Recruiting
Connecticut Children's Medical Center Hartford Connecticut Recruiting
Rare Disease Research FL LLC Kissimmee Florida Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
University of Iowa Stead Family Children's Hospital Iowa City Iowa Recruiting
The Johns Hopkins Hospital Baltimore Maryland Not Yet Recruiting
Boston Children's Hospital Boston Massachusetts Recruiting
The Curators of the University of Missouri on behalf of University of Missouri Health Care Columbia Missouri Not Yet Recruiting
Columbia University Herbert Irving Comprehensive Cancer Center New York New York Recruiting
Rare Disease Research NC, LLC Hillsborough North Carolina Recruiting
Children's Hospital Philadelphia - Neurology Philadelphia Pennsylvania Not Yet Recruiting
Neurology Rare Disease Center Flower Mound Texas Recruiting
Texas Children's Hospital Houston Texas Recruiting
Childrens Hospital of The Kings Daughters Norfolk Virginia Not Yet Recruiting
Sydney Children's Hospital, Randwick Randwick New South Wales Recruiting
Royal Children's Hospital Parkville Victoria Not Yet Recruiting
University of Calgary - Alberta Children's Hospital Calgary Alberta Not Yet Recruiting
BC Children's Hospital Vancouver British Columbia Not Yet Recruiting
The Hospital for Sick Children Toronto Ontario Not Yet Recruiting
Addenbrooke's Hospital Cambridge Cambridgeshire Recruiting
Great Ormond Street Hospital for Children London Greater London Recruiting
MDUK Oxford Neuromuscular Centre Oxford Oxfordshire Not Yet Recruiting
Sheffield Children's Hospital Sheffield South Yorkshire Recruiting
Royal Hospital for Children Glasgow Strathclyde Recruiting
Birmingham Heartlands Hospital Birmingham West Midlands Recruiting
Leeds General Infirmary Leeds West Yorkshire Recruiting

More argenx trials in Australia

A Prospective Longitudinal Study in Adults With Multifocal Motor Neuropathy Active, not recruiting
A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia Active, not recruiting
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis Recruiting
A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy Active, not recruiting
ARGX-117 in Deceased Donor Kidney Transplant Recipients at Risk for Delayed Graft Function Active, not recruiting

Other trials for the same condition

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy Active, not recruiting
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07287982 on ClinicalTrials.gov ↗ ← All trials in Australia