Ultrasound-driven Stratification in CIDP: A Prospective Observational Study Integrating Imaging and Circulating Biomarkers
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 30
- 主要终点
- Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
时间窗: 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.
次要结局
- Clinical response status(Month 6 and Month 12)
- Clinical response status - Hand Grip Strength(Month 6 and Month 12)
- Clinical response status - Medical Research Council (MRC) Sum Score(Month 6 and Month 12)
- Clinical response status - Inflammatory Rasch-built Overall Disability Scale (I-RODS)(Months 6 and 12)
- Clinical response status - Timed Up and Go (TUG)(Month 6 and Month 12)
- Clinical response status - Pain Visual Analog Scale (VAS)(Months 6 and 12)
- Clinical response status - Patient Global Impression of Severity (PGI-S)(Months 6 and 12)
- Clinical response status - Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP)(Months 6 and 12)
- Association between biomarkers and ultrasound phenotypes(Baseline, Month 3, Month 6, and Month 12)
- Association between biomarkers and clinical severity(Baseline, Month 3, Month 6, and Month 12)
- Association between biomarkers and refractory disease(Baseline, Month 3, Month 6, and Month 12)