Condition: Spinal Muscular Atrophy (SMA) · Sponsor: argenx
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 description comes directly from the study's public registry record.
Sabine Coppieters, MD · 857-350-4834 · Clinicaltrials@argenx.com
Always discuss trial participation with your own doctor first.
| Arkansas Children's Hospital | Little Rock, Arkansas, United States | Recruiting |
| Rady Childrens Hospital | San Diego, California, United States | Not Yet Recruiting |
| Stanford University Medical Center | Stanford, California, United States | Not Yet Recruiting |
| Connecticut Children's Medical Center | Hartford, Connecticut, United States | Recruiting |
| Rare Disease Research FL LLC | Kissimmee, Florida, United States | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| University of Iowa Stead Family Children's Hospital | Iowa City, Iowa, United States | Not Yet Recruiting |
| The Johns Hopkins Hospital | Baltimore, Maryland, United States | Not Yet Recruiting |
| Boston Children's Hospital | Boston, Massachusetts, United States | Not Yet Recruiting |
| The Curators of the University of Missouri on behalf of University of Missouri Health Care | Columbia, Missouri, United States | Not Yet Recruiting |
| Columbia University Herbert Irving Comprehensive Cancer Center | New York, New York, United States | Not Yet Recruiting |
| Rare Disease Research NC, LLC | Hillsborough, North Carolina, United States | Recruiting |
+ 5 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT07287982