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.

Study Overview

• Status: Not yet recruiting
• Conditions: Chronic Inflammation; CIDP; Demyelinating Polyneuropathy
• Intervention / Treatment: Other: Standard-of-care treatment for CIDP; Other: Refractory CIDP

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

• Study Contact: Name: Abderhmane Slioui; Phone Number: +33 0492038953; Email: slioui.a@chu-nice.fr
• Study Contact Backup: Name: Angela Puma; Phone Number: +33 0492035435; Email: puma.ar@chu-nice.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Other: Standard-of-care treatment for 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.
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.	Other: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	month 3

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical response status	Clinical response status assessed in Group 1 using predefined response categories	Month 6 and Month 12
Clinical response status - Hand Grip Strength	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	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)	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)	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)	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)	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)	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	statistical analysis of association of biological elements	Baseline, Month 3, Month 6, and Month 12
Association between biomarkers and clinical severity	statistical analysis of association of biological and clinical elements	Baseline, Month 3, Month 6, and Month 12
Association between biomarkers and refractory disease	statistical analysis of association of biological elements	Baseline, Month 3, Month 6, and Month 12

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Nice

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): September 30, 2029

Study Registration Dates

• First Submitted: July 17, 2026
• First Submitted That Met QC Criteria: July 17, 2026
• First Posted (Actual): July 22, 2026

Study Record Updates

• Last Update Posted (Actual): July 22, 2026
• Last Update Submitted That Met QC Criteria: July 17, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Polyneuropathies; Polyradiculoneuropathy; Pathological Conditions, Signs and Symptoms; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
• Other Study ID Numbers: 26Neuro03

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No