A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)

clinicaltrials.gov/study/NCT06078553

Is a

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Clinical study

Clinical Study attributes

NCT Number

NCT060785530

Health Conditions in Trial

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Congenital myasthenic syndrome

Trial Recruitment Size

300

Trial Sponsor

argenx

Clinical Trial Start Date

2023

Primary Completion Date

2025

Study Completion Date

2025

Clinical Trial Study Type

Observational0

Observational Clinical Trial Type

Cohort0

Observational Study Perspective

Other0

Official Name

Multicenter, Multinational, Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes0

Last Updated

October 12, 2023

Study summary

Participants will attend up to 3 study visits and complete home digital physical activity monitoring for 1 week after each visit. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design.

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A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)

Contents

Clinical Study attributes

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