MedTrace Logo

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

NCT06436742 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-04-23

No results posted yet for this 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.

Conditions

  • Congenital Myasthenic Syndrome
  • CMS

Interventions

BIOLOGICAL

ARGX-119

Intravenous infusion of ARGX-119

OTHER

Placebo

Intravenous infusion of placebo

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-24
Primary Completion
2027-08-24
Completion
2028-01-24
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Italy
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06436742 on ClinicalTrials.gov