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

Recruitingn/aAges 2 Years - anyAllargenx
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In plain words

What the trial is, and whether it could suit you

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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Can understand the requirements of the study and can provide written informed consent/assent, and willingness and ability to comply with the study protocol procedures * Is male or female and aged โ‰ฅ2 years at the time of providing informed consent/assent * Has a diagnosis of CMS due to biallelic pathogenic mutations in DOK7 or any pathogenic mutations in MUSK, AGRN, or LRP4 * Has a total Quantitative Myasthenia Gravis (QMG) score of โ‰ฅ3 (applies only to participants aged โ‰ฅ6 years) * For participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine), participant must have been receiving the medication for โ‰ฅ3 months before screening/baseline Exclusion Criteria: * Known medical condition that would interfere with an accurate assessment of CMS, in the investigator's opinion * Is currently participating in any interventional clinical study with a study drug at the time of providing informed consent/assent * Diagnosis of CMS due to mutation of any gene other than DOK7, MUSK, AGRN, or LRP4
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What it tests

  • Not listed
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Where

33 sites
UC Davis Health - UC Davis Health Midtown Ambulatory Care Center
Sacramento, United States
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, United States
Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital
O'Fallon, United States
Harvard Medical School - Boston Children's Hospital
Boston, United States
Mayo Clinic
Rochester, United States
Ohio State Martha Moorehouse
Columbus, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, United States
Medical University of Vienna
Vienna, Austria

โ€ฆand 25 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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