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

Study Identifier:
ARGX-119-24-SMA-2001
ClinicalTrials.gov Identifier:
NCT07287982
EudraCT Identifier:
N/A
EU CT Number:
2025-523496-32-00
Sponsor:
argenx
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Biological: ARGX-119 IV
Date
Dec 2025 - May 2029
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 5 - 17 Years
Requirements Information
Inclusion and 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.

Protocol 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).

Trial Locations

Location
Status
Location
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Status
Recruiting
Location
Rady Childrens Hospital
San Diego, California, United States, 92123
Status
Not yet recruiting
Location
Stanford University Medical Center
Stanford, California, United States, 94305
Status
Not yet recruiting
Location
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
Status
Not yet recruiting
Location
Rare Disease Research FL LLC
Kissimmee, Florida, United States, 34746
Status
Recruiting
Location
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Status
Not yet recruiting
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