- ICH GCP
- Registre américain des essais cliniques
- Essai clinique 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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Abderhmane Slioui
- Numéro de téléphone: +33 0492038953
- E-mail: slioui.a@chu-nice.fr
Sauvegarde des contacts de l'étude
- Nom: Angela Puma
- Numéro de téléphone: +33 0492035435
- E-mail: puma.ar@chu-nice.fr
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
Méthode d'échantillonnage
Population étudiée
La description
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 d'étude
Comment l'étude est-elle conçue ?
Détails de conception
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
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.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
Délai: 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
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Clinical response status
Délai: 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
Délai: Month 6 and Month 12
|
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
Délai: 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)
Délai: 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
|
Months 6 and 12
|
|
Clinical response status - Timed Up and Go (TUG)
Délai: Month 6 and Month 12
|
Timed Up and Go (TUG) test performed in Group 1 to evaluate functional mobility - measure in seconds
|
Month 6 and Month 12
|
|
Clinical response status - Pain Visual Analog Scale (VAS)
Délai: Months 6 and 12
|
Pain intensity assessed in Group 1 with Visual Analog Scale (VAS) from 0 to 10
|
Months 6 and 12
|
|
Clinical response status - Patient Global Impression of Severity (PGI-S)
Délai: 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)
Délai: 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
|
|
Association between biomarkers and ultrasound phenotypes
Délai: Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological elements
|
Baseline, Month 3, Month 6, and Month 12
|
|
Association between biomarkers and clinical severity
Délai: Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological and clinical elements
|
Baseline, Month 3, Month 6, and Month 12
|
|
Association between biomarkers and refractory disease
Délai: Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological elements
|
Baseline, Month 3, Month 6, and Month 12
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies du système nerveux
- Processus pathologiques
- Maladies neuromusculaires
- Maladie chronique
- Attributs de la maladie
- Maladies auto-immunes
- Maladies du système immunitaire
- Maladies du système nerveux périphérique
- Maladies auto-immunes du système nerveux
- Maladies démyélinisantes
- Polyneuropathies
- Polyradiculonévrite
- Conditions pathologiques, signes et symptômes
- Polyradiculonévrite Chronique Inflammatoire Démyélinisante
Autres numéros d'identification d'étude
- 26Neuro03
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
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