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

Conditions

• Spinal Muscular Atrophy (SMA)

Eligibility Criteria

Sex

ALL

Age

5 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• ARGX-119 IV — BIOLOGICAL
  Intravenous infusion of ARGX-119
• Placebo IV — OTHER
  Intravenous infusion of placebo

Study Details

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).

Key Dates

Start date

Dec 19, 2025

Status verified

Apr 2026

Primary completion

May 28, 2029

Completion

May 28, 2029

Study Design

Enrollment

60 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: DBTP - ARGX-119 IV
  Participants receive ARGX-119 IV during the DBTP
• Placebo Comparator: DBTP - Placebo IV
  Participants receive placebo IV during the DBTP
• Placebo Comparator: ATEP - ARGX-119 IV
  Participants receive ARGX-119 IV during the ATEP. Participants from ARGX-119 IV arm in the DBTP will receive placebo once to maintain the DBTP blinding

Primary Outcome Measure

Incidence of AEs [ Time Frame: Up to 124 weeks ]

Central Contacts

• Sabine Coppieters, MD
  857-350-4834
  [email protected]

Locations (17)

Find similar trials in Little Rock, AR

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