Ultrasound-driven Stratification in CIDP

N/ANot Yet RecruitingNCT07719153

Centre Hospitalier Universitaire de Nice30 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

CIDP Chronic Inflammation Demyelinating Polyneuropathy

Interventions

Standard-of-care treatment for CIDPOTHER

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.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 90 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1

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.

Time frame: month 3

Secondary Outcomes

Clinical response status

Clinical response status assessed in Group 1 using predefined response categories

Time frame: Month 6 and Month 12

Clinical response status - Hand Grip Strength

Hand Grip Strength assessed in Group 1 using a dynamometer. measure in kg

Time frame: Month 6 and Month 12

Clinical response status - Medical Research Council (MRC) Sum Score

Medical Research Council (MRC) Sum Score assessed in Group 1 to evaluate global muscle strength. The total MRC score ranges from 0 to 60

Time frame: Month 6 and Month 12

Clinical response status - Inflammatory Rasch-built Overall Disability Scale (I-RODS)

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

Time frame: Months 6 and 12

Clinical response status - Timed Up and Go (TUG)

Timed Up and Go (TUG) test performed in Group 1 to evaluate functional mobility - measure in seconds

Time frame: Month 6 and Month 12

Clinical response status - Pain Visual Analog Scale (VAS)

Pain intensity assessed in Group 1 with Visual Analog Scale (VAS) from 0 to 10

Time frame: Months 6 and 12

Clinical response status - Patient Global Impression of Severity (PGI-S)

Patient Global Impression of Severity (PGI-S) assessed in Group 1. 1-item questionnaire

Time frame: Months 6 and 12

Clinical response status - Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP)

Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP) score assessed in Group 1.6-items

Time frame: Months 6 and 12

Association between biomarkers and ultrasound phenotypes

statistical analysis of association of biological elements

Time frame: Baseline, Month 3, Month 6, and Month 12

Association between biomarkers and clinical severity

statistical analysis of association of biological and clinical elements

Time frame: Baseline, Month 3, Month 6, and Month 12

Association between biomarkers and refractory disease

statistical analysis of association of biological elements

Time frame: Baseline, Month 3, Month 6, and Month 12

Ultrasound-driven Stratification in CIDP

Overview

Conditions

Interventions

Eligibility

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts