Retinal DegenerationVQDRNCT06185517
Validation of Questionnaire in Retinal Degeneration
A study testing Questionnaire translation and validation in people with Retinal Degeneration, run by Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts.
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Retinal Degeneration
- What is being tested?
- Questionnaire translation and validation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 135 participants
- Where
- No sites listed yet
- Study dates
- Jan 2024to Jan 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
No sites are listed yet.
About this study
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.
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.
Read the 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
- Conditions
- Retinal Degeneration
- Being tested
- Questionnaire translation and validation
- Medical subject headings
- Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire translation and validationDiagnostic test
Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). Validation of a French translation
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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). Receives: Diagnostic Test: Questionnaire translation and validation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reliability
will be assessed using intra-class correlations (ICC) between questionnaire measures in test-retest.
Measured: 2 months
Acceptability
will be assessed by analyzing a comprehension questionnaire administered to participants.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2023
- Study startJan 2024 (estimated)
- Primary completionJan 2025 (estimated)
- Study completionJan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- None (open label)
- Enrolment
- 135 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
- Sponsor type
- University, hospital or other
- Collaborators
- Street Lab
- Responsible party
- Sponsor
- Investigators
- Isabelle AUDO, Principal investigator, Centre Hospitalier National d'Ophtalmologie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06185517
- Sponsor's protocol ID
- P22-07
- Acronym
- VQDR
- Status last verified
- Jun 2023
- First submitted
- 16 Jun 2023
- Last updated
- 29 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT06185517
- Acronym / sponsor ID
- VQDR / P22-07
- Conditions
- Retinal Degeneration
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 135 participants (estimated)
- Lead sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-VingtsUniversity, hospital or other
- Collaborators
- Street Lab
- Study start
- Jan 2024 (estimated)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jan 2025 (estimated)
- First posted
- 29 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Comprehension Phase | Experimental | Diagnostic Test: Questionnaire translation and validationAfter 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. |
| Validation Phase | Experimental | Diagnostic Test: Questionnaire translation and validationThe aim is to validate the French translation of the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Questionnaire translation and validationMichigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). Validation of a French translation | Diagnostic test | Comprehension Phase, Validation Phase |
Primary outcome
Reliability
Time frame: 2 months
will be assessed using intra-class correlations (ICC) between questionnaire measures in test-retest.
Acceptability
Time frame: 2 months
will be assessed by analyzing a comprehension questionnaire administered to participants.
Internal validity,
Time frame: 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.
Construct validity
Time frame: 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.
Sensitivity
Time frame: 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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Isabelle AUDO principal investigator
Centre Hospitalier National d'Ophtalmologie
Questions about this trial
What is this clinical trial studying?
A study testing Questionnaire translation and validation in people with Retinal Degeneration, run by Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Nabil BROUK (+331 40 02 11 26, nbrouk@15-20.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reliability (time frame: 2 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Dec 2023.