Clinical Evaluation of Sensory Neuronopathies: the Neuronoscore Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 70
- 试验地点
- 19
- 主要终点
- The Clinical Global Impression of Change (CGI-C) and The Patient Global Impression of Change (PGI-C).
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Group experimental
干预措施: Longitudinal monitoring of clinical assessment scores to identify the most appropriate tool for tracking disease progression in sensory neuronopathies. (Other)
结局指标
主要结局
The Clinical Global Impression of Change (CGI-C) and The Patient Global Impression of Change (PGI-C).
时间窗: 6 months and 12 months
Global Impression of change measure evaluating overall change in clinical status compared with baseline.
次要结局
- miSS Score change(6 months and 12 months)
- SEARS change(6 months, 12 months)
- CADT change(6 months and 12 months)
- SARA change(6 months and 12 months)
- ONLS change(6 months and 12 months)
- I-RODS change(6 months and 12 months)
- 9-Hole Peg Test change(6 months and 12 months)
- Timed Up and Go test change(6 months and 12 months)
- Modified Rankin Scale change(6 months and 12 months)
- Quantified Rydel tuning fork test change(6 months and 12 months)
- Visual Analog Scale change(6 months and 12 months)
- Electroneuromyography(6 months and 12 months)