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
Public ClinicalTrials.gov record NCT07287982. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT07287982
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 5 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 18, 2025
- Primary completion
- May 27, 2029
- Completion
- May 27, 2029
- Last update posted
- Aug 26, 2026
2025 – 2029
United States locations
- U.S. sites
- 16
- U.S. states
- 14
- U.S. cities
- 16
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | Recruiting |
| Rady Childrens Hospital | San Diego | California | 92123 | Withdrawn |
| Stanford University Medical Center | Stanford | California | 94305 | Recruiting |
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | Recruiting |
| Rare Disease Research FL LLC | Kissimmee | Florida | 34746 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | 52242 | Recruiting |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21205 | Not yet recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| The Curators of the University of Missouri on behalf of University of Missouri Health Care | Columbia | Missouri | 65212 | Not yet recruiting |
| Columbia University Herbert Irving Comprehensive Cancer Center | New York | New York | 10032 | Not yet recruiting |
| Rare Disease Research NC, LLC | Hillsborough | North Carolina | 27278 | Recruiting |
| Children's Hospital Philadelphia - Neurology | Philadelphia | Pennsylvania | 19104 | Not yet recruiting |
| Neurology Rare Disease Center | Flower Mound | Texas | 75028 | Recruiting |
| Texas Children's Hospital | Houston | Texas | 77030 | Not yet recruiting |
| Childrens Hospital of The Kings Daughters | Norfolk | Virginia | 23507 | Not yet recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 7 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07287982, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 26, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07287982 live on ClinicalTrials.gov.