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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 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.

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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).

Conditions Studied

Spinal Muscular Atrophy (SMA)

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

49 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, categorized as Phase 2. The registered enrollment target is 60 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 271-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (78% lower). The listed sponsor is argenx, which has 49 total studies on file at ClinicalTrials.gov.

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 reports 17 study locations spanning 15 distinct geographic areas - top geographies include 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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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.