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NCT07287982 · ClinicalTrials.gov registry record · Phase 2
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
A Phase 2 study of Spinal Muscular Atrophy (SMA), sponsored by argenx.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 60
- Enrollment target
- 17
- Study locations
NCT07287982: Recruiting Phase 2 study of Spinal Muscular Atrophy (SMA), sponsored by argenx.
NCT07287982 is a Phase 2 study of Spinal Muscular Atrophy (SMA) that is actively recruiting participants, run by argenx. The registered enrollment target is 60 participants, below the 271-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (78% lower). The trial reports 17 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07287982, a Phase 2 study of Spinal Muscular Atrophy (SMA), is actively recruiting participants, sponsored by argenx.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 60 participants
- Enrollment target
- 17
- Study locations
Study 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).
Primary Outcome
Adverse Events
Conditions Studied
Interventions
- OTHER Placebo IV
- BIOLOGICAL ARGX-119 IV
Study Locations (17)
California
- Rady Childrens Hospital - San Diego
- Stanford University Medical Center - Stanford
Texas
- Neurology Rare Disease Center - Flower Mound
- Texas Children's Hospital - Houston
Arkansas
- Arkansas Children's Hospital - Little Rock
Connecticut
- Connecticut Children's Medical Center - Hartford
Florida
- Rare Disease Research FL LLC - Kissimmee
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Iowa
- University of Iowa Stead Family Children's Hospital - Iowa City
Maryland
- The Johns Hopkins Hospital - Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 60 participants |
| Start Date | 2025-12-19 |
| Est. Completion | 2029-05-28 |
| Phase | Phase 2 |
What NCT07287982 shows while recruiting
NCT07287982 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 60 participants, a relatively small participant target, below the 271-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (78% lower).
The record links to 1 condition, with Spinal Muscular Atrophy (SMA) appearing as the primary indexed condition, and to 2 interventions - of which Placebo IV is the first listed.
NCT07287982 lists 17 locations in 15 states (California, Texas, Arkansas).
Frequently Asked Questions
What is clinical trial NCT07287982 about?
NCT07287982 is a clinical study titled "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". 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 partici...
What is the current status of trial NCT07287982?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 60 participants. The study started on 2025-12-19. Estimated completion is 2029-05-28.
What conditions does trial NCT07287982 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy (SMA).
What interventions are being tested in trial NCT07287982?
The interventions under investigation include: Placebo IV (OTHER), ARGX-119 IV (BIOLOGICAL).
Who is sponsoring clinical trial NCT07287982?
This trial is sponsored by argenx, which has 49 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07287982 being conducted?
This trial has 17 study locations across Arkansas, California, Connecticut, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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