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NCT07287982PHASE2Recruiting

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

argenx

Start Date

12/19/2025

Completion Date

5/28/2029

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).

Detailed Description

This phase 2 study aims to establish proof of concept with the age-appropriate dose of ARGX-119 in ambulant pediatric patients with spinal muscular atrophy (SMA). Despite available treatments, there remains an unmet medical need for patients with SMA. Neuromuscular junction (NMJ) dysfunction contributes to the pathophysiology of SMA, including muscle weakness and fatigability. Activation of muscle-specific kinase (MuSK) by ARGX-119 may stabilize and improve NMJ function in patients with SMA, reducing muscle weakness and fatigability, and improving quality of life.

Eligibility Criteria

Age Range: 5 years to 17 years

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.

Interventions

BIOLOGICAL

ARGX-119 IV

OTHER

Placebo IV

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Conditions

Spinal Muscular Atrophy (SMA)

Locations

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

Rady Childrens Hospital

San Diego, California 92123

United States

Stanford University Medical Center

Stanford, California 94305

United States

Connecticut Children's Medical Center

Hartford, Connecticut 06106

United States

Rare Disease Research FL LLC

Kissimmee, Florida 34746

United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois 60611

United States

University of Iowa Stead Family Children's Hospital

Iowa City, Iowa 52242

United States

The Johns Hopkins Hospital

Baltimore, Maryland 21205

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

The Curators of the University of Missouri on behalf of University of Missouri Health Care

Columbia, Missouri 65212

United States

Columbia University Herbert Irving Comprehensive Cancer Center

New York, New York 10032

United States

Rare Disease Research NC, LLC

Hillsborough, North Carolina 27278

United States

Children's Hospital Philadelphia - Neurology

Philadelphia, Pennsylvania 19104

United States

St. Jude Children's Research Hospital

Memphis, Tennessee 38105

United States

Neurology Rare Disease Center

Flower Mound, Texas 75028

United States

Texas Children's Hospital

Houston, Texas 77030

United States

Childrens Hospital of The Kings Daughters

Norfolk, Virginia 23507

United States