Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

RECRUITING 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

NCT07287982 · View on ClinicalTrials.gov ↗

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).

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

FieldValue
Enrollment Target 60 participants
Start Date 2025-12-19
Est. Completion 2029-05-28
Phase Phase 2

Sponsor

argenx

96 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT07287982

The ClinicalTrials.gov registry entry for NCT07287982 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 60 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is argenx, which has 96 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT07287982 reports 17 study locations spanning 15 distinct geographic areas — top geographies include California, Texas, Arkansas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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). These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT07287982?

The interventions under investigation include: Placebo IV (OTHER), ARGX-119 IV (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT07287982?

This trial is sponsored by argenx, which has 96 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial