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- Ensayo clínico NCT05493605
Compromiso cardíaco en la enfermedad de Wilson (WIL-HEART)
Heart damage by copper accumulation has been reported in Wilson's Disease. However, the disease epidemiology is still poorly understood. A number of studies on pediatric populations have not shown any significant cardiac involvement apart from early dysautonomia. This could suggest that the clinical manifestations related to the copper accumulation in the heart appears with the duration of the disease. Case-control studies on adult populations have highlighted various electrocardiographic (ECG) abnormalities more frequent in patients with Wilson's Disease than in healthy volunteers, but all these studies involved small number of patients (maximum 60). The hypothesis is that there is cardiac involvement in Wilson's Disease, requiring screening, follow-up and appropriate support.
Descripción general del estudio
Estado
Condiciones
Tipo de estudio
Inscripción (Estimado)
Fase
- No aplica
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Amélie YAVCHITZ
- Número de teléfono: 01 48 03 64 54
- Correo electrónico: ayavhitz@for.paris
Copia de seguridad de contactos de estudio
- Nombre: Aurélia POUJOIS
- Número de teléfono: 01 48 30 66 56
- Correo electrónico: apoujois@for.paris
Ubicaciones de estudio
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Paris, Francia, 75019
- Reclutamiento
- Fondation Adolphe de Rothschild
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Contacto:
- Aurélia Poujois
- Número de teléfono: 0148036656
- Correo electrónico: apoujois@for.paris
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
- Adult patient with Wilson's disease confirmed by a Leipzig score ≥ 4
- Express consent to participate in the study by the patient or legal guardian in the case of patients under guardianship or by the patient assisted by his curator in the case of patients under guardianship
- Member of or beneficiary of a Social Security scheme
Exclusion Criteria:
- Absolute or relative contraindication to MRI or contrast media
- Pregnant, parturient or breast-feeding women: a urine pregnancy test will be carried out in women of childbearing age
- Patient with hepatic decompensation (Child-Pugh score stage C)
- Patient in neuro-psychiatric decompensation
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Otro
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Otro: Cases
The intervention consists in setting up a morphological and rhythmological cardiological follow-up patients with confirmed Wilson disease. It will require the wearing of a long Holter duration (21 days) and for some patients the installation of an implantable cardiac monitor.The long-time ECG holter is used to record heart rhythm for 21 days and detect possible arrhythmias (accelerations of the heart) or conduction disorders (slowdowns of the heart). |
The intervention consists of setting up a morphological and rhythmological cardiological follow-up of patients with confirmed Wilson's disease. It will require the wearing of a long-term Holter (21 days) and for some patients the installation of an implantable cardiac monitor. |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Contrast-enhanced cardiac MRI - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal contrast-enhanced cardiac MRI results |
Day 0
|
|
Contrast-enhanced cardiac MRI - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) |
Day 0
|
|
Transthoracic echocardiography - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal transthoracic echocardiography results |
Day 0
|
|
Transthoracic echocardiography - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) |
Day 0
|
|
Chest computed tomography scan without contrast - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal coronary artery calcium score Calcium score : The higher the coronary calcium score, the greater the cardiovascular risk. A score of 0 (min) means that no calcium is seen in the heart. A score greater than 300 is a sign of very high to severe disease. |
Day 0
|
|
Electrocardiogram - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal electrocardiogram results |
Day 0
|
|
Electrocardiogram - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) |
Day 0
|
|
Clinical examination - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal clinical examination |
Day 0
|
|
Clinical examination - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) |
Day 0
|
|
Blood and urine tests - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal blood and urine tests results Blood tests performed : lipid profile, glycosylated hemoglobin (HbA1C), cardiac enzymes (troponin C, NT-proBNP), ultra-sensitive C-reactive protein, sodium level, potassium level, urea and creatinine clearance, TSH, fibrinogen Urine test performed : proteinuria |
Day 0
|
|
Blood and urine tests - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) Blood tests performed : lipid profile, glycosylated hemoglobin (HbA1C), cardiac enzymes (troponin C, NT-proBNP), ultra-sensitive C-reactive protein, sodium level, potassium level, urea and creatinine clearance, TSH, fibrinogen Urine test performed : proteinuria |
Day 0
|
|
Lying and standing blood pressure tests - Day 0
Periodo de tiempo: Day 0
|
Percentage of patients with abnormal blood pressure tests results |
Day 0
|
|
Lying and standing blood pressure tests - Day 0
Periodo de tiempo: Day 0
|
Description of abnormalities (frequency and percentage) |
Day 0
|
|
Implantable loop recorder or ECG holter recorder- Day 21
Periodo de tiempo: Day 21
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Percentage of patients with abnormal implantable loop record (or ECG holter record) The device will record from Day 0 to Day 21 Implantable loop recorder assessment will be only performed on patients with syncope. |
Day 21
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Implantable loop recorder or ECG holter recorder- Day 21
Periodo de tiempo: Day 21
|
Description of abnormalities (frequency and percentage) The device will record from Day 0 to Day 21 Implantable loop recorder assessment will be only performed on patients with syncope. |
Day 21
|
|
Transthoracic echocardiography - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal transthoracic echocardiography results |
Year 3
|
|
Transthoracic echocardiography - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) |
Year 3
|
|
Contrast-enhanced cardiac MRI - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal contrast-enhanced cardiac MRI results |
Year 3
|
|
Contrast-enhanced cardiac MRI - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) |
Year 3
|
|
Chest computed tomography scan without contrast - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal coronary artery calcium score Calcium score : The higher the coronary calcium score, the greater the cardiovascular risk. A score of 0 (min) means that no calcium is seen in the heart. A score greater than 300 is a sign of very high to severe disease. |
Year 3
|
|
Electrocardiogram - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal electrocardiogram results |
Year 3
|
|
Electrocardiogram - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) |
Year 3
|
|
Clinical examination - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal clinical examination |
Year 3
|
|
Clinical examination - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) |
Year 3
|
|
Blood and urine tests - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal blood or urine tests results Blood tests performed : lipid profile, glycosylated hemoglobin (HbA1C), cardiac enzymes (troponin C, NT-proBNP), ultra-sensitive C-reactive protein, sodium level, potassium level, urea and creatinine clearance, TSH, fibrinogen Urine test performed : proteinuria |
Year 3
|
|
Blood and urine tests - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) Blood tests performed : lipid profile, glycosylated hemoglobin (HbA1C), cardiac enzymes (troponin C, NT-proBNP), ultra-sensitive C-reactive protein, sodium level, potassium level, urea and creatinine clearance, TSH, fibrinogen Urine test performed : proteinuria |
Year 3
|
|
Lying and standing blood pressure tests - Year 3
Periodo de tiempo: Year 3
|
Percentage of patients with abnormal blood pressure tests results |
Year 3
|
|
Lying and standing blood pressure tests - Year 3
Periodo de tiempo: Year 3
|
Description of abnormalities (frequency and percentage) |
Year 3
|
Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades Cerebrales
- Enfermedades del Sistema Nervioso Central
- Enfermedades del Sistema Nervioso
- Metabolismo, errores congénitos
- Enfermedades Genéticas Congénitas
- Enfermedades metabólicas
- Enfermedades del Sistema Digestivo
- Enfermedades neurodegenerativas
- Enfermedades del HIGADO
- Trastornos del movimiento
- Trastornos Heredodegenerativos, Sistema Nervioso
- Enfermedades de los ganglios basales
- Enfermedades Cerebrales Metabólicas Congénitas
- Enfermedades Cerebrales Metabólicas
- Metabolismo de metales, errores congénitos
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Enfermedades Nutricionales y Metabólicas
- Degeneración hepatolenticular
Otros números de identificación del estudio
- APS_2021_9
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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