Actively Recruiting
A Phase 2 Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous ARGX-119 in Children Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Led by argenx · Updated on 2026-05-22
60
Participants Needed
17
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ARGX-119 for children aged 5 to less than 18 years with spinal muscular atrophy (SMA) in this phase 2 study. The study aims to find the correct dose of ARGX-119 and assess its safety, how it works, how it moves through the body, and how the immune system reacts. The study addresses an unmet need since muscle weakness and fatigue in SMA are linked to neuromuscular junction dysfunction, and ARGX-119 may help stabilize this. Participants will be randomly assigned to receive either intravenous ARGX-119 or a placebo for 24 weeks during a double-blinded treatment period, while continuing their current SMA treatments. After this period, those who complete it can enter an open-label extension lasting about 100 weeks, where all participants will receive ARGX-119 intravenously. During the extension, participants originally on ARGX-119 will receive a placebo once to maintain blinding. Throughout the study, participants will undergo various assessments including safety monitoring for adverse events up to 124 weeks, evaluation of muscle function changes from baseline to week 24, and measurement of ARGX-119 levels in the blood. Researchers will also track immune responses to the drug and muscle endurance using walking tests. The total participation can last over two years with regular evaluations of safety and effectiveness.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 5 to less than 18 years and able to comply with study requirements
- Documented genetic diagnosis of 5q-Spinal Muscular Atrophy
- Receiving stable SMA treatment like nusinersen, risdiplam, or history of onasemnogene abeparvovec
- Able to walk at least 50 meters without walking aids at screening
You will not qualify if you...
- Medical conditions interfering with SMA assessment or increasing risk, as judged by the investigator
- Major surgery within 3 months before screening or planned during the study, except spinal fusion
- Use of antimyostatin therapies within the past 6 months
- Severe scoliosis with curvature over 40 degrees or contractures at screening
- History of spinal fusion within 6 months before screening or planned during the study
- Respiratory insufficiency requiring invasive or noninvasive ventilation while awake
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 weeks
Participants receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period.
Repeated visits for intravenous infusions and assessments during treatment
Duration - Up to 100 weeks after treatment
Participants continue to be monitored for safety, efficacy, and immunogenicity after the treatment period ends.
Periodic visits for assessments and safety monitoring
Trial Site Locations
Total: 17 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Rady Childrens Hospital
San Diego, California, United States, 92123
Not Yet Recruiting
3
Stanford University Medical Center
Stanford, California, United States, 94305
Not Yet Recruiting
4
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
Not Yet Recruiting
5
Rare Disease Research FL LLC
Kissimmee, Florida, United States, 34746
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
Not Yet Recruiting
8
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21205
Not Yet Recruiting
9
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
10
The Curators of the University of Missouri on behalf of University of Missouri Health Care
Columbia, Missouri, United States, 65212
Not Yet Recruiting
11
Columbia University Herbert Irving Comprehensive Cancer Center
New York, New York, United States, 10032
Not Yet Recruiting
12
Rare Disease Research NC, LLC
Hillsborough, North Carolina, United States, 27278
Actively Recruiting
13
Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
14
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Not Yet Recruiting
15
Neurology Rare Disease Center
Flower Mound, Texas, United States, 75028
Actively Recruiting
16
Texas Children's Hospital
Houston, Texas, United States, 77030
Not Yet Recruiting
17
Childrens Hospital of The Kings Daughters
Norfolk, Virginia, United States, 23507
Not Yet Recruiting
Research Team
S
Sabine Coppieters, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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