A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119. The full duration of the study is approximately 38 months.

NCT Number	NCT06436742
Lead Sponsor	argenx
Conditions	Congenital Myasthenic Syndrome, CMS
Enrollment	15 participants
Start Date	2024-09-24
Primary Completion	2027-08-24 (estimated)
Study Completion	2028-01-24 (estimated)
Updated on ClinicalTrials.gov	2026-07-24