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

Condition(s) studied

Investigational drug(s) / intervention(s)

ARGX-119: Intravenous infusion of ARGX-119

Placebo: Intravenous infusion of placebo

Study 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.

Eligibility

Primary outcome measure(s)

• Assessment of adverse events (AEs) — Up to week 42
• Change from active-treatment baseline over time for 6MWT distance — Up to 72 weeks
  The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured.

Trial sites (9)