NCT06078553

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

Multicenter, Multinational, Natural History Study in Participants With Congenital Myasthenic Syndromes Due to Mutations in DOK7, MUSK, AGRN, or LRP4

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 100 (estimated)
• Sponsor: argenx · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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

Timeline

• Start date: 2024-02-13
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2023-10-12
• Last updated: 2026-03-13

Locations

19 sites across 10 countries: United States, Austria, Canada, France, Germany, Italy, Japan, Poland, Spain, United Kingdom