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
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
Interventions
ARGX-119 IV
Placebo IV
Conditions
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
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
Sydney Children's Hospital, Randwick
Randwick, New South Wales 2031
Australia
Royal Children's Hospital
Parkville, Victoria 3052
Australia
University of Calgary - Alberta Children's Hospital
Calgary, Alberta T3B 6A*
Canada
BC Children's Hospital
Vancouver, British Columbia V6H 3V4
Canada
The Hospital for Sick Children
Toronto, Ontario M5G 1X8
Canada
Addenbrooke's Hospital
Cambridge, Cambridgeshire CB2 0QQ
United Kingdom
Great Ormond Street Hospital for Children
London, Greater London WC1N 3JH
United Kingdom
MDUK Oxford Neuromuscular Centre
Oxford, Oxfordshire OX3 9DU
United Kingdom
Sheffield Children's Hospital
Sheffield, South Yorkshire S10 2TH
United Kingdom
Royal Hospital for Children
Glasgow, Strathclyde G51 4TF
United Kingdom
Birmingham Heartlands Hospital
Birmingham, West Midlands B9 5SS
United Kingdom
Leeds General Infirmary
Leeds, West Yorkshire LS1 3EX
United Kingdom