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A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

NCT06078553 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2026-04-27

No results posted yet for this study

Summary

Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4.

More information can be found here: https://clinicaltrials.argenx.com/cms

Conditions

  • Congenital Myasthenic Syndrome

Sponsors & Collaborators

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-13
Primary Completion
2027-06-30
Completion
2027-06-30

Countries

  • United States
  • Austria
  • Canada
  • France
  • Germany
  • Italy
  • Japan
  • Poland
  • 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 NCT06078553 on ClinicalTrials.gov