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RECRUITINGPhase 2INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

Who May Be Eligible (Plain English)

Who May Qualify: - Is aged ≥5 to \<18 years when completing the willing to sign a consent form 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 Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

BIOLOGICAL

ARGX-119 IV

Intravenous infusion of ARGX-119

OTHER

Placebo IV

Intravenous infusion of placebo

Locations (17)

Arkansas Children's Hospital
Little Rock, Arkansas, United States
Rady Childrens Hospital
San Diego, California, United States
Stanford University Medical Center
Stanford, California, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Rare Disease Research FL LLC
Kissimmee, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 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
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
Rare Disease Research NC, LLC
Hillsborough, North Carolina, United States
Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Neurology Rare Disease Center
Flower Mound, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Childrens Hospital of The Kings Daughters
Norfolk, Virginia, United States