Clinical trial NCT06185517: Validation of Questionnaire in Retinal Degeneration
Official title: 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.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Retinal Degeneration
Lead sponsor: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Sponsor class: OTHER
Collaborators: name: Street Lab
First posted: 2023-12-29
Enrollment: 135
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Comprehension phase:

After an initial translation of the questionnaires into French, the aim of this of this phase is to ensure the level of understanding of each and potential suggestions, during a personal or telephone interview.

Validation phase:

The aim is to validate the French translation of the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ), and in particular to assess: internal validity, construct validity, acceptability, reliability, sensitivity, specificity.
Primary purpose: DIAGNOSTIC

Brief summary: 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.

Detailed description: 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

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Reliability
timeFrame: 2 months
measure: Acceptability
timeFrame: 2 months
measure: Internal validity,
timeFrame: 2 months
measure: Construct validity
timeFrame: 2 months
measure: Sensitivity
timeFrame: 2 months
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Centre Hospitalier National d'Ophtalmologie
name: Isabelle AUDO
role: PRINCIPAL_INVESTIGATOR
References: