Validation of Questionnaire in Retinal Degeneration

Retinal Degeneration Clinical Trial

— VQDR  

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.

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06185517
• Other study ID #: P22-07
• Status: Not yet recruiting
• Phase: N/A
• Start date: January 2024
• Est. completion date: January 2025

Study information

• Verified date: June 2023
• Source: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
• Contact: Nabil BROUK
• Phone: +331 40 02 11 26
• Email: nbrouk[@]15-20.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 135
• Est. completion date: January 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Retinal Degeneration

Intervention

Diagnostic Test:
• Questionnaire translation and validation
Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). Validation of a French translation

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts	Street Lab

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reliability	will be assessed using intra-class correlations (ICC) between questionnaire measures in test-retest.	2 months
Primary	Acceptability	will be assessed by analyzing a comprehension questionnaire administered to participants.	2 months
Primary	Internal validity,	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.	2 months
Primary	Construct validity	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.	2 months
Primary	Sensitivity	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.	2 months