Actively Recruiting
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
Led by argenx · Updated on 2026-04-22
60
Participants Needed
17
Research Sites
179 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official 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...
- 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
You will not qualify if you...
- 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Not Yet 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
CONTACT
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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