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Study identifier: NCT06078553 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Congenital Myasthenic Syndrome  ·  Sponsor: argenx

PhaseN/A
Planned participants100
Who can joinAll sexes, 2 Years to no upper limit
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Sabine Coppieters, MD  ·  857-350-4834  ·  clinicaltrials@argenx.com

Always discuss trial participation with your own doctor first.

Locations (32)

UC Davis Health - UC Davis Health Midtown Ambulatory Care CenterSacramento, California, United StatesRecruiting
Ann and Robert H Lurie Children's Hospital of Chicago - Main HospitalChicago, Illinois, United StatesRecruiting
Hospital Sisters Health System (HSHS) - St Elizabeth's HospitalO'Fallon, Illinois, United StatesRecruiting
Harvard Medical School - Boston Children's HospitalBoston, Massachusetts, United StatesRecruiting
Ohio State Martha MoorehouseColumbus, Ohio, United StatesRecruiting
The Children's Hospital of Philadelphia (CHOP)Philadelphia, Pennsylvania, United StatesRecruiting
Medical University of ViennaVienna, AustriaRecruiting
Hopital Universitaire de Bruxelles/ Academisch Ziekenhuis BrusselBrussels, Anderlecht, BelgiumRecruiting
Pseg Centro De Pesquisa ClinicaS.ASão Paulo, BrazilRecruiting
Universidade de Sao Paulo (USP) Hospital das Clinicas da Faculdade de Medicina (HCFMUSP)São Paulo, BrazilRecruiting
The Children's Hospital of Eastern (CHEO), OttawaOttawa, Ontario, CanadaRecruiting
The Ottawa Hospital - Civic CampusOttawa, CanadaRecruiting

+ 20 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06078553