NCT06436742

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

A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Congenital Myasthenic Syndrome
• CMS

Interventions

Type	Name	Description
BIOLOGICAL	ARGX-119	Intravenous infusion of ARGX-119
OTHER	Placebo	Intravenous infusion of placebo

Timeline

• Start date: 2024-09-24
• Primary completion: 2027-08-24
• Completion: 2028-01-24
• First posted: 2024-05-31
• Last updated: 2026-02-06

Locations

9 sites across 6 countries: United States, Canada, France, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study