French Translation and Validation of the Michigan Retinal Degeneration Questionnaire and the Michigan Vision-Related Anxiety Questionnaire in a Cohort of People With Hereditary Retinal Degeneration.
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 135
- 主要终点
- Reliability
研究概览
简要总结
While inherited retinal diseases are well characterized anatomically and functionally, it is also essential to understand their impact on patients' quality of life, from their ability to perform the tasks of daily living to the psychological impact of the disease on patients. Questionnaires (PRO, Patient-Reported Outcomes) are important tools for assessing this impact. However, PROs validated in French are rare, and often poorly adapted to populations with hereditary retinal degeneration. New PROs particularly adapted to retinal degeneration have recently been validated in English: the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). Validation of a French translation will provide a standardized, reliable tool for French-speaking patients, and enable these questionnaires to be used in gene therapy clinical trials, to assess the efficacy of treatments and their impact on patients' quality of life.
详细描述
Interventional research of category 2 with minimal risks and constraints. This is a prospective, monocentric, longitudinal, non-randomized study to validate the translation of questionnaires to assess abilities in daily living tasks and the potential psychological repercussions of visual impairment in various hereditary retinal dystrophies.
The research will be conducted in two distinct phases:
Comprehension phase and validation phase
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female
- •Age: 18 to 80
- •Sufficient knowledge of the French language to ensure understanding of the tasks to be performed and the instructions received.
- •Signed consent after being informed by the investigator.
- •Affiliation with health insurance.
- •Affected by one of the following rare pathologies, with different levels of visual field, acuity and contrast sensitivity impairment:
- •Non-syndromic rod-cone dystrophy (retinopathy pigmentosa, RP): diagnosis confirmed by a specialist.
- •Non-syndromic rod-cone dystrophy: diagnosis confirmed by a specialist.
- •Non-syndromic Stargardt's disease: diagnosis confirmed by a specialist.
- •Non-syndromic congenital night blindness: diagnosis confirmed by a specialist.
- •Non-syndromic achromatopsia: diagnosis confirmed by a specialist.
排除标准
- •Pregnancy
- •Inability to give informed consent.
- •Cataract surgery within 3 months of inclusion.
- •Hearing impairment that may interfere with comprehension during the interview.
- •Functional amblyopia.
- •Inability to comply with instructions to perform study tasks or study visits.
- •Drug treatment that may cause motor, visual or cognitive impairment (PSAs, neuroleptics, etc.) or that may interfere with study assessments.
- •Other uncontrolled ophthalmic conditions that may interfere with evaluation.
- •Participation in another clinical trial that may interfere with the present study.
- •Patient under guardianship, curatorship or legal protection
结局指标
主要结局
Reliability
时间窗: 2 months
will be assessed using intra-class correlations (ICC) between questionnaire measures in test-retest.
Construct validity
时间窗: 2 months
Will be assessed by examining the relationships between questionnaire scores and other theoretically related measures. High correlations with similar measures and low correlations with unrelated measures indicate good construct validity.
Internal validity,
时间窗: 2 months
Internal validity is crucial to ensure that the questionnaire questions correctly measure the concept under study. It is assessed by examining the correlations between similar questions. High correlations indicate good internal validity, which means that the questionnaire is reliable and accurate for assessing the target concept.
Acceptability
时间窗: 2 months
will be assessed by analyzing a comprehension questionnaire administered to participants.
Sensitivity
时间窗: 2 months
Will be assessed by analysing the questionnaire's ability to detect significant differences between groups of participants with different levels of disease severity. A sensitive questionnaire will make it possible to identify subtle differences between groups and will therefore be more useful for assessing the impact of the disease on patients' quality of life.
次要结局
未报告次要终点