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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

Study acronym: Sparkle
StatusRecruiting
PhasePhase 2
Sponsorargenx
Started2025-12-19
View on ClinicalTrials.gov ↗

Amendment history

2026-09-10
minor
Study Status, Contacts/Locations v11
Study sitesdetails revised at 1 of 23 sites
2026-08-25
minor
Study Status, Contacts/Locations v10
Study sites24→23
2026-08-17
minor
Study Status, Contacts/Locations v9
Study sitesdetails revised at 1 of 24 sites
2026-08-06
minor
Study Status, Contacts/Locations v8
Trial sites expanded: 17 → 24 locations
2026-07-20
minor
Study Status, Contacts/Locations v7
Study sitesdetails revised at 1 of 17 sites
Show 6 earlier versions
2026-05-20
minor
Study Status, Contacts/Locations v6
Study sitesdetails revised at 1 of 17 sites
2026-04-17
minor
Study Status, Contacts/Locations v5
Study sitesdetails revised at 1 of 17 sites
2026-03-16
minor
Study Status, Contacts/Locations v4
Study sitescontacts updated at 1 of 17 sites
2026-02-25
minor
Study Status, Contacts/Locations v3
Study sites15→17
2026-01-21
minor
Study Status, Contacts/Locations v2
Study sites2→15
2026-01-16
notable
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Study sites0→2
2025-12-15
minor
Original filing
56 trials monitored
See 2 trials at risk
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).
Trial Details
NCT Number NCT07287982
Lead Sponsor argenx
Conditions Spinal Muscular Atrophy (SMA)
Enrollment 60 participants
Start Date 2025-12-19
Primary Completion 2029-05-28 (estimated)
Study Completion 2029-05-28 (estimated)
Updated on ClinicalTrials.gov 2026-09-14