- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT07719153
Ultrasound-driven Stratification in CIDP (TAILOR-CIDP)
Ultrasound-driven Stratification in CIDP: A Prospective Observational Study Integrating Imaging and Circulating Biomarkers
Chronic inflammatory demyelinating polyradiculoneuritis (CIDP) is a rare autoimmune neuropathy characterized by significant clinical and therapeutic heterogeneity. Despite the availability of effective treatments, the response to intravenous immunoglobulins remains highly variable, and there are currently no validated biomarkers that can predict this response.
At the same time, high-resolution nerve ultrasound now makes it possible to identify different morphological profiles that may reflect distinct pathophysiological mechanisms.
This prospective, observational, single-center study, conducted at the Nice University Hospital, aims to determine whether nerve ultrasound profiles are associated with therapeutic response, clinical severity, and various biomarkers in the blood and cerebrospinal fluid. It includes two predefined cohorts: 20 patients with newly diagnosed PIDC, enrolled before the initiation of immunomodulatory treatment (Group 1), and 10 patients with refractory PIDC and clinically significant disability despite adequate prior treatment (Group 2). The ultimate goal is to develop a stratification strategy that will enable more personalized care for patients with PIDC.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Abderhmane Slioui
- Número de teléfono: +33 0492038953
- Correo electrónico: slioui.a@chu-nice.fr
Copia de seguridad de contactos de estudio
- Nombre: Angela Puma
- Número de teléfono: +33 0492035435
- Correo electrónico: puma.ar@chu-nice.fr
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Método de muestreo
Población de estudio
Descripción
Inclusion Criteria:
- Male or female aged 18 years or older.
- Diagnosis of CIDP according to the 2021 EAN/PNS criteria; eligible phenotypes include typical CIDP, asymmetric CIDP (MADSAM/Lewis-Sumner syndrome), and pure motor CIDP. Pure sensory CIDP is excluded.
- Ability to undergo protocol assessments, including clinical evaluation, electrophysiological studies, nerve ultrasound, and blood sampling.
- Ability to provide written informed consent.
- Affiliation with a health insurance system or equivalent.
Group 1-specific criteria:
- Newly diagnosed CIDP.
- No previous immunomodulatory treatment for CIDP before baseline study assessment.
- Planned initiation of IVIg according to standard clinical practice.
Group 2-specific criteria:
- Established CIDP with persistent clinically relevant disability.
- Documented inadequate, partial, transient, or absent response despite adequate prior therapy, according to the final refractory disease definition.
- Stable treatment exposure before inclusion according to the final protocol.
Exclusion Criteria:
- Pure sensory CIDP.
- Alternative cause of neuropathy, including hereditary, metabolic, toxic, or other acquired neuropathies judged to better explain the clinical picture.
- Motor neuron disease, myopathy, neuromuscular junction disorder, or another neurological or neuromuscular condition interfering with clinical, electrophysiological, or ultrasound interpretation.
- CIDP mimic or alternative diagnosis.
- Active infection likely to influence study assessments.
- Active malignancy or other major systemic condition likely to confound biomarker interpretation.
- Concomitant autoimmune or inflammatory disease likely to materially influence cytokine or complement measurements.
- Severe psychiatric or cognitive disorder interfering with participation.
- Participation in another interventional trial when incompatible with the present protocol.
- Inability or unwillingness to comply with study procedures.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
|
Treatment-naïve newly diagnosed CIDP
Participants will receive treatment according to routine clinical practice.
In Group 1, first-line treatment will usually consist of intravenous immunoglobulin (IVIg), with subsequent therapeutic decisions made by the treating neurologist according to clinical response and standard care.
No investigational intervention is assigned by the study protocol.
|
Participants will receive treatment according to routine clinical practice.
In Group 1, first-line treatment will usually consist of intravenous immunoglobulin (IVIg), with subsequent therapeutic decisions made by the treating neurologist according to clinical response and standard care.
No investigational intervention is assigned by the study protocol.
|
|
Refractory CIDP
Participants with refractory CIDP will continue or receive treatments according to routine clinical practice.
The study does not assign or modify treatment and is limited to observational phenotyping and biomarker analyses.
|
Participants with refractory CIDP will continue or receive treatments according to routine clinical practice.
The study does not assign or modify treatment and is limited to observational phenotyping and biomarker analyses.
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
Periodo de tiempo: month 3
|
Clinical response status at Month 3 after initiation of first-line IVIg in treatment-naïve patients with newly diagnosed chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
Response categories will be predefined and may include remission, responder, partial responder, and non-responder, based primarily on adjusted INCAT and Hand Grip Strength.
|
month 3
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Clinical response status
Periodo de tiempo: Month 6 and Month 12
|
Clinical response status assessed in Group 1 using predefined response categories
|
Month 6 and Month 12
|
|
Clinical response status - Hand Grip Strength
Periodo de tiempo: Month 6 and Month 12
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Hand Grip Strength assessed in Group 1 using a dynamometer.
measure in kg
|
Month 6 and Month 12
|
|
Clinical response status - Medical Research Council (MRC) Sum Score
Periodo de tiempo: Month 6 and Month 12
|
Medical Research Council (MRC) Sum Score assessed in Group 1 to evaluate global muscle strength.
The total MRC score ranges from 0 to 60
|
Month 6 and Month 12
|
|
Clinical response status - Inflammatory Rasch-built Overall Disability Scale (I-RODS)
Periodo de tiempo: Months 6 and 12
|
Inflammatory Rasch-built Overall Disability Scale (I-RODS) score assessed in Group 1 to evaluate to evaluate activity- 24 items, score from 0 to 48
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Months 6 and 12
|
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Clinical response status - Timed Up and Go (TUG)
Periodo de tiempo: Month 6 and Month 12
|
Timed Up and Go (TUG) test performed in Group 1 to evaluate functional mobility - measure in seconds
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Month 6 and Month 12
|
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Clinical response status - Pain Visual Analog Scale (VAS)
Periodo de tiempo: Months 6 and 12
|
Pain intensity assessed in Group 1 with Visual Analog Scale (VAS) from 0 to 10
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Months 6 and 12
|
|
Clinical response status - Patient Global Impression of Severity (PGI-S)
Periodo de tiempo: Months 6 and 12
|
Patient Global Impression of Severity (PGI-S) assessed in Group 1. 1-item questionnaire
|
Months 6 and 12
|
|
Clinical response status - Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP)
Periodo de tiempo: Months 6 and 12
|
Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP) score assessed in Group 1.6-items
|
Months 6 and 12
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Association between biomarkers and ultrasound phenotypes
Periodo de tiempo: Baseline, Month 3, Month 6, and Month 12
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statistical analysis of association of biological elements
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Baseline, Month 3, Month 6, and Month 12
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Association between biomarkers and clinical severity
Periodo de tiempo: Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological and clinical elements
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Baseline, Month 3, Month 6, and Month 12
|
|
Association between biomarkers and refractory disease
Periodo de tiempo: Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological elements
|
Baseline, Month 3, Month 6, and Month 12
|
Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
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 del Sistema Nervioso
- Procesos Patológicos
- Enfermedades Neuromusculares
- Enfermedad crónica
- Atributos de la enfermedad
- Enfermedades autoinmunes
- Enfermedades del sistema inmunológico
- Enfermedades del Sistema Nervioso Periférico
- Enfermedades Autoinmunes del Sistema Nervioso
- Enfermedades desmielinizantes
- Polineuropatías
- Polirradiculoneuropatía
- Condiciones Patológicas, Signos y Síntomas
- Polirradiculoneuropatía Crónica Inflamatoria Desmielinizante
Otros números de identificación del estudio
- 26Neuro03
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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