Validation of Questionnaire in Retinal Degeneration

NCT ID: NCT06185517

Last Updated: 2023-12-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

135 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-01-31

Study Completion Date

2025-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Retinal Degeneration

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Comprehension Phase

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.

Group Type EXPERIMENTAL

Questionnaire translation and validation

Intervention Type DIAGNOSTIC_TEST

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

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).

Group Type EXPERIMENTAL

Questionnaire translation and validation

Intervention Type DIAGNOSTIC_TEST

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Questionnaire translation and validation

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

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Street Lab

UNKNOWN

Sponsor Role collaborator

Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Isabelle AUDO

Role: PRINCIPAL_INVESTIGATOR

Centre Hospitalier National d'Ophtalmologie

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Nabil BROUK

Role: CONTACT

Phone: +331 40 02 11 26

Email: [email protected]

Hayet SERHANE

Role: CONTACT

Phone: +331 40 02 11 44

Email: [email protected]

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

P22-07

Identifier Type: -

Identifier Source: org_study_id