Myotonic Dystrophy 1GoodDiagNMDNCT06270186
Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach
A study testing Type 1 myotonic dystrophy in people with Myotonic Dystrophy 1, run by Centre Hospitalier Universitaire de Nice.
Trial summary
Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that primarily affects muscle tissue, resulting in myotonia (difficulty relaxing after contraction) and atrophy (progressive muscle weakening with decreased muscle volume). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- Type 1 myotonic dystrophy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Nov 2023to Nov 2024
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- male or female age ≥ 18
- suffering with type 1 myotonic dystrophy confirmed by molecular biology
- suffering with dysexecutive impairments, with a pathologic BREF score ≤ 15
- affiliated to social security
- able to understand the inform consent form
Who cannot join
- suffering with visual or auditive impairments preventing them doing tests
- suffering with other pathologies preventing them doing tests
- suffering with motor impairments preventing them holding joysticks or carrying helmet
- protection by law under guardianship, or who cannot participate in a clinical study under Article L. 1121-16 of the French Code of Public Health
- patient under treatment that may affect cognitive functions (ie : Modafinil)
- participation in the last 3 months in a clinical research study in which he / she has been exposed to a pharmaceutical product or a medical device
- pregnant or breastfeeding female patient
About this study
Official title: Feasibility of a Virtual Reality Approach in the Assessment of Executive Functions in Patients With Type 1 Myotonic Dystrophy : Pilot Study
Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that primarily affects muscle tissue, resulting in myotonia (difficulty relaxing after contraction) and atrophy (progressive muscle weakening with decreased muscle volume). It also affects eyes, heart, endocrine system, gastrointestinal system and central nervous system. Specific cognitive abilities are impaired in patients with MD1 such as attention, visio-spatial or visio-building abilities as well as executive dysfunctions.
Currently, the cognitive assessment of MD1 patients is based on classical neuropsychological tests, which are time-consuming and require a MD1 expert neuropsychologist. Moreover, it is usually very difficult for MD1 patients to accept performing these tests, and when they agree to perform them, they usually give up before the end. This finding is more frequent in MD1 patients with high level of cognitive impairment.
Read more
In order to overcome these difficulties in assessing cognitive functions of MD1 patients, the investigators decided to use innovative tools such as virtual reality, which allow individuals to experience a sensory-motor and cognitive experience in a digitally world through a helmet, glasses and joysticks.
The start-up My Cyber Royaume from Lille, in collaboration with the reference center of neuromuscular diseases from Nice coordinated by Pr Sacconi, have developed a software "Good Diag NMD" which uses virtual reality to assess cognitive disorders, more specifically executive functions in patients with type 1 myotonic dystrophy.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- Type 1 myotonic dystrophy
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Type 1 myotonic dystrophyDiagnostic test
To compare classical neuropsychological test and on "Good Diag NMD" software in Type 1 myotonic dystrophy patients
| Study group | Role | What they receive |
|---|---|---|
| Type 1 myotonic dystrophy | Other | Type 1 myotonic dystrophy patients doing classical neuropsychological test and on "Good Diag NMD" software Receives: Diagnostic Test: Type 1 myotonic dystrophy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the feasibility of the use "Good Diag NMD" software in the evaluation of cognitive executive dysfunctions in patients with dystrophy myotonia 1
The feasibility "Good Diag NMD" software in patient with type 1 myotonic dystrophy (DM1) will be determined as the rate of patients having completed the Good Diag NMD session in full. Satisfactory feasibility is considered if this rate of patients is greater or equal to 50%.
Measured: 0 months
Secondary outcomes (4)
Establish a possible relationship between the genotype of DM1 patients and the scores obtained during "Good Diag NMD" session
Genotype of DM1 patients will be determined by molecular biology technique, ie the size of GTC triplet expansion will indicate the of severity of the genetic abnormality . Then, we will analyse a possible correlation of the genotype with the scores obtained during "Good Diag NMD" session.
Measured: 0 months
To compare the satisfaction score of type 1 myotonic dystrophy patients between classic neuropsychological tests and the Good Diag NMD session
The satisfaction will be measured with a satisfaction questionnaire called "Assessment questionnaire" (value between 0 to 50)
Measured: 0 months
Compare the scores of "Good Diag NMD" session (scores of perseveration, inhibition and flexibility) with the scores of the classical neuropsychological tests
The following correlations will be examined studied:
- The level of correlation between perseveration score (0-149) and Frontal Efficiency Rapid Battery (BREF) score (0-18)
- The perseveration score (0-149) and Short Cognitive Battery (B2C) score
- The inhibition score (0-77) and interference score (score I) from Stroop test (-30 and +30)
- flexibility score (0-119) and completion time of Trail Making Test (TMT) B
Measured: 0 months
Establish a possible correlation between the severity of the disease, the severity of muscular impairment and the scores of Good Diag NMD session
Measured: 0 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2024
- Study startNov 2023 (estimated)
- Primary completionNov 2024 (estimated)
- Study completionNov 2024 (estimated)
Why it stopped: study withdrawn by the sponsor for logistical reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sabrina SACCONI, Principal investigator, Centre Hospitalier Universitaire de Nice
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06270186
- Sponsor's protocol ID
- 20-AOIP-04
- Acronym
- GoodDiagNMD
- Status last verified
- Feb 2024
- First submitted
- 14 Jan 2022
- Last updated
- 21 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility of a Virtual Reality Approach in the Assessment of Executive Functions in Patients With Type 1 Myotonic Dystrophy : Pilot Study
- ClinicalTrials.gov ID
- NCT06270186
- Acronym / sponsor ID
- GoodDiagNMD / 20-AOIP-04
- Conditions
- Myotonic Dystrophy 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: study withdrawn by the sponsor for logistical reasons
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Nov 2023 (estimated)
- Primary completion
- Nov 2024 (estimated)
- Study completion
- Nov 2024 (estimated)
- First posted
- 21 Feb 2024 (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.
Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that primarily affects muscle tissue, resulting in myotonia (difficulty relaxing after contraction) and atrophy (progressive muscle weakening with decreased muscle volume). It also affects eyes, heart, endocrine system, gastrointestinal system and central nervous system. Specific cognitive abilities are impaired in patients with MD1 such as attention, visio-spatial or visio-building abilities as well as executive dysfunctions.
Currently, the cognitive assessment of MD1 patients is based on classical neuropsychological tests, which are time-consuming and require a MD1 expert neuropsychologist. Moreover, it is usually very difficult for MD1 patients to accept performing these tests, and when they agree to perform them, they usually give up before the end. This finding is more frequent in MD1 patients with high level of cognitive impairment.
In order to overcome these difficulties in assessing cognitive functions of MD1 patients, the investigators decided to use innovative tools such as virtual reality, which allow individuals to experience a sensory-motor and cognitive experience in a digitally world through a helmet, glasses and joysticks.
The start-up My Cyber Royaume from Lille, in collaboration with the reference center of neuromuscular diseases from Nice coordinated by Pr Sacconi, have developed a software "Good Diag NMD" which uses virtual reality to assess cognitive disorders, more specifically executive functions in patients with type 1 myotonic dystrophy.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Type 1 myotonic dystrophy | Other | Diagnostic Test: Type 1 myotonic dystrophyType 1 myotonic dystrophy patients doing classical neuropsychological test and on "Good Diag NMD" software |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- male or female age ≥ 18
- suffering with type 1 myotonic dystrophy confirmed by molecular biology
- suffering with dysexecutive impairments, with a pathologic BREF score ≤ 15
- affiliated to social security
- able to understand the inform consent form
Key exclusion criteria (7 listed)
- suffering with visual or auditive impairments preventing them doing tests
- suffering with other pathologies preventing them doing tests
- suffering with motor impairments preventing them holding joysticks or carrying helmet
- protection by law under guardianship, or who cannot participate in a clinical study under Article L. 1121-16 of the French Code of Public Health
- patient under treatment that may affect cognitive functions (ie : Modafinil)
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 |
|---|---|---|
| Type 1 myotonic dystrophyTo compare classical neuropsychological test and on "Good Diag NMD" software in Type 1 myotonic dystrophy patients | Diagnostic test | Type 1 myotonic dystrophy |
Primary outcome
Evaluate the feasibility of the use "Good Diag NMD" software in the evaluation of cognitive executive dysfunctions in patients with dystrophy myotonia 1
Time frame: 0 months
The feasibility "Good Diag NMD" software in patient with type 1 myotonic dystrophy (DM1) will be determined as the rate of patients having completed the Good Diag NMD session in full. Satisfactory feasibility is considered if this rate of patients is greater or equal to 50%.
4 secondary outcomes
Establish a possible relationship between the genotype of DM1 patients and the scores obtained during "Good Diag NMD" session
Time frame: 0 months
To compare the satisfaction score of type 1 myotonic dystrophy patients between classic neuropsychological tests and the Good Diag NMD session
Time frame: 0 months
Compare the scores of "Good Diag NMD" session (scores of perseveration, inhibition and flexibility) with the scores of the classical neuropsychological tests
Time frame: 0 months
Establish a possible correlation between the severity of the disease, the severity of muscular impairment and the scores of Good Diag NMD session
Time frame: 0 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2024.
- Individual participant dataSharing planned: No
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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.
Sabrina SACCONI principal investigator
Centre Hospitalier Universitaire de Nice
Questions about this trial
What is this clinical trial studying?
A study testing Type 1 myotonic dystrophy in people with Myotonic Dystrophy 1, run by Centre Hospitalier Universitaire de Nice.
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 5 inclusion and 7 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: withdrawn, as of the sponsor's last update on 21 Feb 2024. 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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the feasibility of the use "Good Diag NMD" software in the evaluation of cognitive executive dysfunctions in patients with dystrophy myotonia 1 (time frame: 0 months). 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 21 Feb 2024.