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).
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
60
Intravenous infusion of ARGX-119
Intravenous infusion of placebo
Arkansas Children's Hospital
Little Rock, Arkansas, United States
NOT_YET_RECRUITINGRady Childrens Hospital
San Diego, California, United States
NOT_YET_RECRUITINGStanford University Medical Center
Stanford, California, United States
NOT_YET_RECRUITINGConnecticut Children's Medical Center
Hartford, Connecticut, United States
NOT_YET_RECRUITINGRare Disease Research FL LLC
Kissimmee, Florida, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGUniversity of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States
NOT_YET_RECRUITINGThe Johns Hopkins Hospital
Baltimore, Maryland, United States
NOT_YET_RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
NOT_YET_RECRUITINGThe Curators of the University of Missouri on behalf of University of Missouri Health Care
Columbia, Missouri, United States
NOT_YET_RECRUITING...and 7 more locations
Incidence of AEs
Adverse Events
Time frame: Up to 124 weeks
Incidence of SAEs
Serious Adverse Events
Time frame: Up to 124 weeks
Change in RHS total score from baseline to week 24 of the double blinded treatment period (DBTP)
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)
Time frame: Up to 24 weeks
Change from baseline over time for the 6MWT - distance and fatigue index
In the 6MWT (6-minute walk test) participants will be instructed to walk as fast as possible without jogging or running along a 25- meter linearly marked course for 6 minutes without using walking devices or assistance. Distance walked over the entire 6-minute period, and other parameters, will be determined
Time frame: Up to 124 weeks
ARGX-119 serum concentrations over time
ARGX-119 serum concentrations over time
Time frame: Up to 124 weeks
Incidence of antidrug antibodies (ADA) against ARGX-119
Incidence of antidrug antibodies (ADA) against ARGX-119
Time frame: Up to 124 weeks
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