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Ultrasound-driven Stratification in CIDP (TAILOR-CIDP)

2026年7月17日 更新者: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.

研究概览

研究类型

观察性的

注册 (估计的)

30

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习联系方式

研究联系人备份

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

取样方法

非概率样本

研究人群

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.

描述

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.

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

队列和干预

团体/队列
干预/治疗
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.

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
大体时间: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

次要结果测量

结果测量
措施说明
大体时间
Clinical response status
大体时间: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
大体时间: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
大体时间: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)
大体时间: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)
大体时间: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)
大体时间: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)
大体时间: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)
大体时间: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
大体时间: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
大体时间: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
大体时间:Baseline, Month 3, Month 6, and Month 12
statistical analysis of association of biological elements
Baseline, Month 3, Month 6, and Month 12

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年9月1日

初级完成 (估计的)

2028年12月31日

研究完成 (估计的)

2029年9月30日

研究注册日期

首次提交

2026年7月17日

首先提交符合 QC 标准的

2026年7月17日

首次发布 (实际的)

2026年7月22日

研究记录更新

最后更新发布 (实际的)

2026年7月22日

上次提交的符合 QC 标准的更新

2026年7月17日

最后验证

2026年7月1日

更多信息

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不

研究美国 FDA 监管的设备产品

不

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