Ultrasound-driven Stratification in CIDP

Official title

Ultrasound-driven Stratification in CIDP: A Prospective Observational Study Integrating Imaging and Circulating Biomarkers

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.

Interventions

• 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.
• 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.

Primary outcome measures

• Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1 [Time frame: month 3]

Secondary outcome measures (11)

• Clinical response status [Time frame: Month 6 and Month 12]
• Clinical response status - Hand Grip Strength [Time frame: Month 6 and Month 12]
• Clinical response status - Medical Research Council (MRC) Sum Score [Time frame: Month 6 and Month 12]
• Clinical response status - Inflammatory Rasch-built Overall Disability Scale (I-RODS) [Time frame: Months 6 and 12]
• Clinical response status - Timed Up and Go (TUG) [Time frame: Month 6 and Month 12]
• Clinical response status - Pain Visual Analog Scale (VAS) [Time frame: Months 6 and 12]
• Clinical response status - Patient Global Impression of Severity (PGI-S) [Time frame: Months 6 and 12]
• Clinical response status - Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP) [Time frame: Months 6 and 12]
• Association between biomarkers and ultrasound phenotypes [Time frame: Baseline, Month 3, Month 6, and Month 12]
• Association between biomarkers and clinical severity [Time frame: Baseline, Month 3, Month 6, and Month 12]
• Association between biomarkers and refractory disease [Time frame: Baseline, Month 3, Month 6, and Month 12]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07719153 · 26Neuro03

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗