Validation of Questionnaire in Retinal Degeneration

NCT06185517 | Unknown

• 1 other identifier: P22-07
• Study Type: interventional
• Target: 135
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Retinal Degeneration

Outcome Measures

Primary Outcomes (5)

• Reliability

  will be assessed using intra-class correlations (ICC) between questionnaire measures in test-retest.

  2 months

• Acceptability

  will be assessed by analyzing a comprehension questionnaire administered to participants.

  2 months

• 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

• 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

• 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

Study Arms (2)

Comprehension Phase

EXPERIMENTAL

After an initial translation of the questionnaires into French, the aim of this phase is to ascertain the level of understanding of each questionnaire item and any suggestions, during a telephone or face-to-face interview.

Diagnostic Test: Questionnaire translation and validation

Validation Phase

EXPERIMENTAL

The aim is to validate the French translation of the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ).

Diagnostic Test: Questionnaire translation and validation

Interventions

Questionnaire translation and validation DIAGNOSTIC_TEST

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

Comprehension Phase; Validation Phase

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Pregnancy
• Inability to give informed consent.
• 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

Sponsors & Collaborators

• Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts: lead
• Street Lab: collaborator

MeSH Terms

Conditions

Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Study Officials

• Isabelle AUDO

  Centre Hospitalier National d'Ophtalmologie

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nabil BROUK

CONTACT

+331 40 02 11 26; nbrouk@15-20.fr

Hayet SERHANE

CONTACT

+331 40 02 11 44; hserhane@15-20.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 16, 2023
• First Posted: December 29, 2023
• Study Start: January 1, 2024
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: December 29, 2023

Record last verified: 2023-06