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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
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| Measure | Description | Time Frame |
|---|---|---|
| Summary statistics of retrospective and prospective collection of data on diagnosis. | Up to 12 months | |
| Summary statistics of retrospective and prospective collection of data on health care utilization. | Up to 12 months | |
| Summary statistics of retrospective and prospective collection of data on medications. | Up to 12 months | |
| Summary statistics of retrospective and prospective collection of data on change in health status related to CMS | Up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline for QMG total score, each component score, and raw values | The Quantitative Myasthenia Gravis (QMG) consists of 13 items that assess ocular, bulbar, and limb function. Six of the 13 items are timed endurance tests measured in seconds. Each item has a possible score from 0 to 3, with 3 being the most severe. The total possible score is 39, with higher scores indicating more severe impairments. |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4 receiving standard of care at their treating physician
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sabine Coppieters, MD | Contact | 857-350-4834 | clinicaltrials@argenx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis Health - UC Davis Health Midtown Ambulatory Care Center | Recruiting | Sacramento | California | 95816 | United States |
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| Label | URL |
|---|---|
| Study website | View source |
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| Up to 12 months |
| Change from baseline for MG-ADL total score | The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item scale that assesses MG symptoms and their effects on daily activities. The 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. | Up to 12 months |
| Change from baseline for PROMIS-GHS | The Patient-Reported Outcomes Measurement Information System Global Health Scale (PROMIS-GHS) is a quality-of-life questionnaire that comprises questions on overall physical health, physical function, pain, and fatigue (GPH); and quality of life, mental health, satisfaction with social activities, and emotional problems (GMH). The participant marks their response on a 5-point Likert scale, with lower scores indicating poorer health | Up to 12 months |
| Change from baseline for PROMIS-DFL | The Patient-Reported Outcomes Measurement Information System Dyspnea Functional Limitations (PROMIS-DFL) is a questionnaire that evaluates the impact of dyspnea on the ability to perform daily activities within the last 7 days. The participant marks their response on a 4-point scale (0 to 3), with lower values indicating less functional impairment. | Up to 12 months |
| Change from baseline for EQ-5D-5L | The EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status for clinical and economic appraisal. The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Participants mark their health status from 0 (the worst health you can imagine) to 100 (the best health you can imagine). | Up to 12 months |
| Change from baseline for 3TUG | The Triple Timed Up and Go (3TUG) is an objective measure of mobility requiring 3 repetitions (laps) to assess lower extremity weakness and fatigability. | Up to 12 months |
| Change from baseline for Neuro-QoL Fatigue score | The Quality of Life in Neurological Disorders (Neuro-QoL) Fatigue questionnaire will be completed by participants aged ≥18 years. The Neuro-QoL Pediatric Fatigue questionnaire will be completed by pediatric participants aged ≥12 to <18 years. Participants aged <8 years will be evaluated using the proxy version of the questionnaire. | Up to 12 months |
| Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital | Recruiting | Chicago | Illinois | 60611 | United States |
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| Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital | Recruiting | O'Fallon | Illinois | 62269 | United States |
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| Harvard Medical School - Boston Children's Hospital | Recruiting | Boston | Massachusetts | 02115 | United States |
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| Ohio State Martha Moorehouse | Recruiting | Columbus | Ohio | 43221 | United States |
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| The Children's Hospital of Philadelphia (CHOP) | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| Medical University of Vienna | Recruiting | Vienna | 1090 | Austria |
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| Hopital Universitaire de Bruxelles/ Academisch Ziekenhuis Brussel | Recruiting | Brussels | Anderlecht | 1070 | Belgium |
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| Pseg Centro De Pesquisa ClinicaS.A | Recruiting | São Paulo | 04038-002 | Brazil |
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| The Children's Hospital of Eastern (CHEO), Ottawa | Recruiting | Ottawa | Ontario | K1H8L1 | Canada |
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| The Ottawa Hospital - Civic Campus | Recruiting | Ottawa | K1Y 4E9 | Canada |
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| CHU Bordeaux - Groupe Hospitalier Pellegrin - Neurology | Recruiting | Bordeaux | 33076 | France |
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| Hospices Civils de Lyon (HCL) - Hopital Pierre Wertheimer - Neurology | Recruiting | Bron | 69500 | France |
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| CHU Lille Hopital Salengro - Neurology | Recruiting | Lille | 59037 | France |
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| CHU Timone | Recruiting | Marseille | 13385 | France |
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| Assistance Publique Hopitaux de Paris - Hopital Pitie-Salpetriere | Recruiting | Paris | 75013 | France |
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| Centre Hospitalier Regional Universitaire de Strasbourg - Hopital de Hautepierre | Recruiting | Strasbourg | 67000 | France |
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| Charite - Universitaetsmedizin Berlin | Recruiting | Berlin | 10117 | Germany |
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| UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH | Recruiting | Bochum | 44789 | Germany |
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| Universitaetsklinikum Essen (AoR) | Recruiting | Essen | 45147 | Germany |
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| Fondazione IRCCS Istituto Neurologico Carlo Besta | Recruiting | Milan | 20133 | Italy |
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| National Center Hospital, National Center of Neurology and Psychiatry | Recruiting | Kodaira | 187-8551 | Japan |
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| National Hospital Organization Akita Hospital - Neurology | Recruiting | Yurihonjō | 018-1393 | Japan |
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| Medicover Integrated Clinical Services (MICS) Centrum Medyczne Bydgoszcz | Recruiting | Bydgoszcz | 85-065 | Poland |
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| Centrum Medyczne Neurologia Slaska - Neurology | Recruiting | Katowice | 40-689 | Poland |
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| Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego | Active, not recruiting | Warsaw | 02-097 | Poland |
| Hospital Sant Joan de Deu - Pediatric Neurology | Recruiting | Esplugues de Llobregat | 08950 | Spain |
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| Hospital Universitario 12 de Octubre | Recruiting | Madrid | 28041 | Spain |
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| Hospital Universitari i Politecnic La Fe | Recruiting | Valencia | 46026 | Spain |
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| Ulster Hospital | Recruiting | Dundonald | BT16 1RH | United Kingdom |
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| Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital | Recruiting | Oxford | OX3 9DU | United Kingdom |
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| ID | Term |
|---|---|
| D020294 | Myasthenic Syndromes, Congenital |
| ID | Term |
|---|---|
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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