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 · NCT07287982
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 5–17 · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 2
- Target enrollment
- 60
- US sites recruiting
- 11
- Study start
- December 19, 2025
- Record last updated
- October 8, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study 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).
Being studied: ARGX-119 IV; Placebo IV
Conditions: Spinal Muscular Atrophy (SMA)
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
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.
Recruiting US locations
Arkansas Children's Hospital
Little Rock, AR 72202
Site contact: Steven Giompoletti · (501) 364-1430 · giompolettis@archildrens.org
Stanford University Medical Center
Stanford, CA 94305
Site contact: Lidia Choniawko · (650) 304-8094 · lidiacho@stanford.edu
Connecticut Children's Medical Center
Hartford, CT 06106
Site contact: Gyula Acsadi · (860) 545-9460 · gacsadi@connecticutchildrens.org
Rare Disease Research FL LLC
Kissimmee, FL 34746
Site contact: Maria Cordero · (407) 545-7610 · maria.cordero@rarediseaseresearch.com
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, IL 60611
Site contact: Alka Maheshwari · (815) 444-6362 · amaheshwari@luriechildrens.org
University of Iowa Stead Family Children's Hospital
Iowa City, IA 52242
Site contact: Katherine Mathews · (319) 356-1851 · katherine-mathews@uiowa.edu
Boston Children's Hospital
Boston, MA 02115
Site contact: Samantha Remis · (617) 355-2752 · samantha.remis@childrens.harvard.edu
Columbia University Herbert Irving Comprehensive Cancer Center
New York, NY 10032
Site contact: Claudia Chiriboga · (646) 426-3876 · cac3@cumc.columbia.edu
Rare Disease Research NC, LLC
Hillsborough, NC 27278
Site contact: Norma Davis · (470) 665-3051 · norma.davis@rarediseaseresearch.com
Neurology Rare Disease Center
Flower Mound, TX 75028
Site contact: Natalie Gomez · (972) 999-1011 · Natalie.Gomez@Neuromdcenter.com
Texas Children's Hospital
Houston, TX 77030
Site contact: Timothy Lotze · (832) 822-1779 · tlotze@bcm.edu
Study contacts (from the official record)
- Sabine Coppieters, MD · 857-350-4834 · Clinicaltrials@argenx.com