Clinical Evaluation of Sensory Neuronopathies: the Neuronoscore Study

Обзор

Sensory neuronopathies (SN) are a group of rare neuropathies characterized by selective destruction of sensory neurons located in the dorsal root ganglia. SN may result from a wide range of etiologies, particularly paraneoplastic, autoimmune, toxic, and genetic causes. The functional prognosis of patients with SN is generally poor: in a recent study, two-thirds of patients had a modified Rankin Scale (mRS) score ≥3 and nearly half had an mRS ≥4. The absence of reliable biomarkers in neuropathies justifies the use of clinical scales as indicators of disease severity, disability, and treatment response. However, none of the currently available "general neuropathy" scales have been specifically designed or validated for SN. The only scale developed specifically for SN is the SEARS (Sensory Ataxia Rating Scale), proposed in 2019, but it has not been widely used nor validated in large populations. As a result, the absence of a clinical scale specifically designed for patients with SN makes longitudinal follow-up more challenging, particularly when assessing the response to immunomodulatory or immunosuppressive treatments when these therapies are indicated.

Вмешательства

• Другое Longitudinal monitoring of clinical assessment scores to identify the most appropriate tool for tracking disease progression in sensory neuronopathies.
  After patient consent, three follow-up visits (T0, T6, T12) will be scheduled. Some information will already be collected at baseline (demographics, medical history, comorbidities). Three visits will be conducted: (T0), Follow-up at 6 months (T6), Follow-up at 12 months (T12) At each visit the following will be assessed: Clinical scales: mISS, SEARS, CADT, SARA, ONLS, I-RODS, 9-Hole Peg Test, Timed Up and Go test, mRS, Quantified Rydel tuning fork test, Visual Analog Scale (VAS) ENMG including

Первичные конечные точки

• The Clinical Global Impression of Change (CGI-C) and The Patient Global Impression of Change (PGI-C). [Срок оценки: 6 months and 12 months]

Вторичные конечные точки (12)

• miSS Score change [Срок оценки: 6 months and 12 months]
• SEARS change [Срок оценки: 6 months, 12 months]
• CADT change [Срок оценки: 6 months and 12 months]
• SARA change [Срок оценки: 6 months and 12 months]
• ONLS change [Срок оценки: 6 months and 12 months]
• I-RODS change [Срок оценки: 6 months and 12 months]
• 9-Hole Peg Test change [Срок оценки: 6 months and 12 months]
• Timed Up and Go test change [Срок оценки: 6 months and 12 months]
• Modified Rankin Scale change [Срок оценки: 6 months and 12 months]
• Quantified Rydel tuning fork test change [Срок оценки: 6 months and 12 months]
• Visual Analog Scale change [Срок оценки: 6 months and 12 months]
• Electroneuromyography [Срок оценки: 6 months and 12 months]

Критерии участия

Критерии включения

• Patient affiliated with or beneficiary of a social security system
• Patient having received appropriate study information
• Adult patient ≥18 years old, male or female
• Patient diagnosed with probable SN according to Camdessanché et al. diagnostic criteria
• SN with one of the following etiologies:

Paraneoplastic SN with anti-Hu or anti-CV2/CRMP5 antibodies SN associated with Sjögren's syndrome, systemic lupus erythematosus, or primary biliary cholangitis Platinum-salt-induced SN SN caused by CANVAS syndrome

Критерии исключения

• Patient unable to understand or read French
• Patient refusal to participate
• Patient known to have another neuropathy phenotype and/or etiology that could significantly influence clinical scales and electrophysiological parameters, including:

• Diabetes mellitus
• Significant alcohol consumption
• Severe chronic kidney disease (GFR <30 ml/min)
• Vitamin B12 and/or vitamin E deficiency
• Vitamin B6 excess
• Chemotherapy other than platinum salts
• HIV infection

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Другое

Центры проведения

Франция · 16 центров

• Chu D Angers — Angers
• Chu de Clermont Ferrand — Clermont-Ferrand
• Chu de Grenoble — Grenoble
• Chu de Lille — Lille
• Chu de Limoges — Limoges
• Hospices Civils de Lyon — Lyon
• Hopitaux Universitaire de Marseille — Marseille
• Chu de Nancy — Nancy
• … и ещё 8 центров

Швейцария · 3 центра

• Hopitaux Universitaire de Geneve — Geneva
• Chuv Centre Hospitalier Vaudois — Lausanne
• Hopital de Sion — Sion

Идентификаторы

NCT: NCT07651540 · 26CH090 · 2026-A00695-46

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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