ARGX-119 for Spinal Muscular Atrophy

(Sparkle Trial)

Recruiting at 1 trial location

Overseen By Sabine Coppieters, MD

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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).

Are You a Good Fit for This Trial?

This trial is for children aged 5 to less than 18 with Spinal Muscular Atrophy (SMA). They must have a genetic diagnosis of SMA, be on stable treatment (nusinersen or risdiplam), or have had onasemnogene abeparvovec. Participants need to walk at least 50 meters unaided in a test.

Inclusion Criteria

I am between 5 and 18 years old and can follow the study rules.

I meet the specific requirements listed.

I have a genetic diagnosis of 5q-SMA.

What Are the Treatments Tested in This Trial?

Interventions

• ARGX-119

Trial Overview

The study tests ARGX-119 IV against a placebo in kids with SMA while they continue their standard treatments. It's double-blinded for the first part, meaning neither doctors nor patients know who gets what. Afterward, all get ARGX-119 in an open-label extension.

How Is the Trial Designed?

Group I: DBTP - ARGX-119 IV Experimental Treatment 1 Intervention

Participants receive ARGX-119 IV during the DBTP

Group II: DBTP - Placebo IV Placebo Group 1 Intervention

Participants receive placebo IV during the DBTP

Group III: ATEP - ARGX-119 IV Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

argenx

Lead Sponsor

Trials

76

Recruited

11,500+

Tim Van Hauwermeiren

argenx

Chief Executive Officer since 2008

B.Sc. and M.Sc. in Bioengineering from Ghent University, Executive MBA from The Vlerick School of Management

Dr. Peter Ulrichts

argenx

Chief Medical Officer since 2023

MD from Maastricht University, PhD in Molecular Immunology from Maastricht University