ARGX-119 IV: Intravenous infusion of ARGX-119
Placebo IV: Intravenous infusion of placebo
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).
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | Recruiting |
| Rady Childrens Hospital | San Diego | California | Withdrawn |
| Stanford University Medical Center | Stanford | California | Recruiting |
| Connecticut Children's Medical Center | Hartford | Connecticut | Recruiting |
| Rare Disease Research FL LLC | Kissimmee | Florida | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | Recruiting |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | Recruiting |
| The Johns Hopkins Hospital | Baltimore | Maryland | Not Yet Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | Recruiting |
| The Curators of the University of Missouri on behalf of University of Missouri Health Care | Columbia | Missouri | Not Yet Recruiting |
| Columbia University Herbert Irving Comprehensive Cancer Center | New York | New York | Not Yet Recruiting |
| Rare Disease Research NC, LLC | Hillsborough | North Carolina | Recruiting |
| Children's Hospital Philadelphia - Neurology | Philadelphia | Pennsylvania | Not Yet Recruiting |
| Neurology Rare Disease Center | Flower Mound | Texas | Recruiting |
| Texas Children's Hospital | Houston | Texas | Recruiting |
| Childrens Hospital of The Kings Daughters | Norfolk | Virginia | Not Yet Recruiting |
| Addenbrooke's Hospital | Cambridge | Cambridgeshire | Recruiting |
| Great Ormond Street Hospital for Children | London | Greater London | Not Yet Recruiting |
| MDUK Oxford Neuromuscular Centre | Oxford | Oxfordshire | Not Yet Recruiting |
| Sheffield Children's Hospital | Sheffield | South Yorkshire | Recruiting |
| Royal Hospital for Children | Glasgow | Strathclyde | Recruiting |
| Birmingham Heartlands Hospital | Birmingham | West Midlands | Recruiting |
| Leeds General Infirmary | Leeds | West Yorkshire | Recruiting |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07287982 on ClinicalTrials.gov ↗ ← All trials in the UK