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
Study identification
- NCT ID
- NCT07287982
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- argenx
- Industry
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Interventions
- ARGX-119 IV Biological
- Placebo IV Other
Biological · Other
Eligibility (public fields only)
- Age range
- 5 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Not listed
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
- Apr 21, 2026
2025 – 2029
United States locations
- U.S. sites
- 17
- U.S. states
- 15
- U.S. cities
- 17
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | Recruiting |
| Rady Childrens Hospital | San Diego | California | 92123 | Not yet recruiting |
| Stanford University Medical Center | Stanford | California | 94305 | Not yet recruiting |
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | Not yet recruiting |
| Rare Disease Research FL LLC | Kissimmee | Florida | 34746 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Not yet recruiting |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | 52242 | Not yet recruiting |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21205 | Not yet recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Not yet 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 |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | 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.
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 Apr 21, 2026 · Synced Apr 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07287982 live on ClinicalTrials.gov.