A Phase 2 Double-Blinded, Randomized, Placebo-Controlled 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
Status: Recruiting
Location: See all (15) locations...
Intervention Type: Other, Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 17
Healthy Volunteers: f
View:
• 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
Locations
United States
Arkansas
Arkansas Children's Hospital
NOT_YET_RECRUITING
Little Rock
California
Rady Childrens Hospital
NOT_YET_RECRUITING
San Diego
Stanford University Medical Center
NOT_YET_RECRUITING
Stanford
Connecticut
Connecticut Children's Medical Center
NOT_YET_RECRUITING
Hartford
Florida
Rare Disease Research FL LLC
NOT_YET_RECRUITING
Kissimmee
Iowa
University of Iowa Stead Family Children's Hospital
NOT_YET_RECRUITING
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
NOT_YET_RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
NOT_YET_RECRUITING
Boston
Missouri
The Curators of the University of Missouri on behalf of University of Missouri Health Care
NOT_YET_RECRUITING
Columbia
North Carolina
Rare Disease Research NC, LLC
RECRUITING
Hillsborough
Pennsylvania
Children's Hospital Philadelphia - Neurology
NOT_YET_RECRUITING
Philadelphia
Tennessee
St. Jude Children's Research Hospital
NOT_YET_RECRUITING
Memphis
Texas
Neurology Rare Disease Center
RECRUITING
Flower Mound
Texas Children's Hospital
NOT_YET_RECRUITING
Houston
Virginia
Childrens Hospital of The Kings Daughters
NOT_YET_RECRUITING
Norfolk
Contact Information
Primary
Sabine Coppieters, MD
Clinicaltrials@argenx.com
857-350-4834
Time Frame
Start Date:2025-12-19
Estimated Completion Date:2029-05-28
Participants
Target number of participants:60
Treatments
Experimental: DBTP - ARGX-119 IV
Participants receive ARGX-119 IV during the DBTP
Placebo_comparator: DBTP - Placebo IV
Participants receive placebo IV during the DBTP
Placebo_comparator: ATEP - ARGX-119 IV
Participants receive ARGX-119 IV during the ATEP. Participants from ARGX-119 IV arm in the DBTP will receive placebo once to maintain the DBTP blinding