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.
調査の概要
状態
研究の種類
入学 (推定)
連絡先と場所
研究連絡先
- 名前:Abderhmane Slioui
- 電話番号:+33 0492038953
- メール:slioui.a@chu-nice.fr
研究連絡先のバックアップ
- 名前:Angela Puma
- 電話番号:+33 0492035435
- メール:puma.ar@chu-nice.fr
参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
サンプリング方法
調査対象母集団
説明
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.
研究計画
研究はどのように設計されていますか?
デザインの詳細
コホートと介入
グループ/コホート |
介入・治療 |
|---|---|
|
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.
|
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Clinical response status to first-line intravenous immunoglobulin (IVIg) for group 1
時間枠: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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Clinical response status
時間枠: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
時間枠: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
時間枠: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)
時間枠: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)
時間枠:Month 6 and Month 12
|
Timed Up and Go (TUG) test performed in Group 1 to evaluate functional mobility - measure in seconds
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Month 6 and Month 12
|
|
Clinical response status - Pain Visual Analog Scale (VAS)
時間枠:Months 6 and 12
|
Pain intensity assessed in Group 1 with Visual Analog Scale (VAS) from 0 to 10
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Months 6 and 12
|
|
Clinical response status - Patient Global Impression of Severity (PGI-S)
時間枠: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)
時間枠: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
|
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Association between biomarkers and ultrasound phenotypes
時間枠:Baseline, Month 3, Month 6, and Month 12
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statistical analysis of association of biological elements
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Baseline, Month 3, Month 6, and Month 12
|
|
Association between biomarkers and clinical severity
時間枠: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
時間枠:Baseline, Month 3, Month 6, and Month 12
|
statistical analysis of association of biological elements
|
Baseline, Month 3, Month 6, and Month 12
|
協力者と研究者
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- 26Neuro03
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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