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Ultrasound-driven Stratification in CIDP (TAILOR-CIDP)

17 de julho de 2026 atualizado por: Centre Hospitalier Universitaire de Nice

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.

Visão geral do estudo

Tipo de estudo

Observacional

Inscrição (Estimado)

30

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Estude backup de contato

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Método de amostragem

Amostra Não Probabilística

População do estudo

The study population will consist of 30 consecutive adult patients with CIDP followed at the Peripheral Nervous System and Muscle Department of CHU Nice. The cohort will include 20 treatment-naïve patients with newly diagnosed CIDP enrolled before initiation of immunomodulatory therapy and 10 patients with established refractory CIDP and persistent clinically relevant disability despite adequate prior treatment. All participants will be assessed within the standard diagnostic and therapeutic care pathway for CIDP.

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

Coortes e Intervenções

Grupo / Coorte
Intervenção / Tratamento
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.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
Prazo: 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 resultados secundários

Medida de resultado
Descrição da medida
Prazo
Clinical response status
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

1 de setembro de 2026

Conclusão Primária (Estimado)

31 de dezembro de 2028

Conclusão do estudo (Estimado)

30 de setembro de 2029

Datas de inscrição no estudo

Enviado pela primeira vez

17 de julho de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

17 de julho de 2026

Primeira postagem (Real)

22 de julho de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

22 de julho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

17 de julho de 2026

Última verificação

1 de julho de 2026

Mais Informações

Termos relacionados a este estudo

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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