RecruitingNCT06078553

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

Sponsor

argenx

Enrollment

100 participants

Start Date

Feb 13, 2024

Study Type

OBSERVATIONAL

Conditions

Congenital Myasthenic Syndrome

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

Eligibility

Min Age: 2 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a new treatment for people with congenital myasthenic syndrome. The study is currently recruiting participants at 32 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(32)

UC Davis Health - UC Davis Health Midtown Ambulatory Care Center

Sacramento, California, United States

Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

Chicago, Illinois, United States

Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital

O'Fallon, Illinois, United States

Harvard Medical School - Boston Children's Hospital

Boston, Massachusetts, United States

Ohio State Martha Moorehouse

Columbus, Ohio, United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Medical University of Vienna

Vienna, Austria

Hopital Universitaire de Bruxelles/ Academisch Ziekenhuis Brussel

Brussels, Anderlecht, Belgium

Pseg Centro De Pesquisa ClinicaS.A

São Paulo, Brazil

Universidade de Sao Paulo (USP) Hospital das Clinicas da Faculdade de Medicina (HCFMUSP)

São Paulo, Brazil

The Children's Hospital of Eastern (CHEO), Ottawa

Ottawa, Ontario, Canada

The Ottawa Hospital - Civic Campus

Ottawa, Canada

CHU Bordeaux - Groupe Hospitalier Pellegrin - Neurology

Bordeaux, France

Hospices Civils de Lyon (HCL) - Hopital Pierre Wertheimer - Neurology

Bron, France

CHU Lille Hopital Salengro - Neurology

Lille, France

CHU Timone

Marseille, France

Assistance Publique Hopitaux de Paris - Hopital Pitie-Salpetriere

Paris, France

Centre Hospitalier Regional Universitaire de Strasbourg - Hopital de Hautepierre

Strasbourg, France

Charite - Universitaetsmedizin Berlin

Berlin, Germany

UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH

Bochum, Germany

Universitaetsklinikum Essen (AoR)

Essen, Germany

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

National Center Hospital, National Center of Neurology and Psychiatry

Kodaira, Japan

National Hospital Organization Akita Hospital - Neurology

Yurihonjō, Japan

Medicover Integrated Clinical Services (MICS) Centrum Medyczne Bydgoszcz

Bydgoszcz, Poland

Centrum Medyczne Neurologia Slaska - Neurology

Katowice, Poland

Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego

Warsaw, Poland

Hospital Sant Joan de Deu - Pediatric Neurology

Esplugues de Llobregat, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Ulster Hospital

Dundonald, United Kingdom

Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital

Oxford, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT06078553

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