Prader-Willi SyndromePRACOM1NCT04526379
Study of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative Remediation
A study testing Questionnaires and t-VNS in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 1 site in France.
Trial summary
The present project project is divided in two parts. The primary aim of the part 1 of this study is to evaluate emotional control abilities of children with Prader-Willi syndrome (PWS) aged from 9 to 15 years and to study repercussions of this supposed lack of abilities on cognitive capacities and behavioral troubles. Read more about this study
- Who is it for?
- Ages 9 to 15 yearsHealthy volunteers can take part
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Questionnaires and t-VNS
- Type of treatment
- Medical device, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteFrance
- Study dates
- 7 Sep 2020to 27 Apr 2022
- 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 9 to 15 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Age from 9 to 15 years old
- Prader-Willi syndrome with identified genotype.
- No psychiatric disorder neither PWS.
- Severe global symptomatology attested by a CGI-S score
Who cannot join
- Identified psychiatric or behavioral disorders
- Severe visual or hearing impairment.
- Sleep apnea syndrome treated with non-invasive ventilation,
- Epileptic seizures
- Cardiac disorders.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Communication in Prader-Willi Syndrome: Study of Emotional Control Related to Behavioral Disorders, Their Daily Repercussions and Examination of an Innovative Therapy: Transcutaneous Electrical Nerve Stimulation of the Vagus Nerve - PRACOM1
The present project project is divided in two parts. The primary aim of the part 1 of this study is to evaluate emotional control abilities of children with Prader-Willi syndrome (PWS) aged from 9 to 15 years and to study repercussions of this supposed lack of abilities on cognitive capacities and behavioral troubles. The study also evaluate influence of the emotional symptomatology of patients on quality of parents' life and on the care of parents and scholar/institutional caregivers. In the second part of this study, the study evaluate the feasibility and the tolerance to a non-invasive device supposed to reduce emotional symptoms in this disease.
Read the detailed description
Prader-Willi Syndrome (PWS) is a rare genetic disorder involving a variety of clinical, behavioral and cognitive symptoms. 83 to 97% of patients have episodes of temper tantrum, associated with an important emotional lability. These different cognitive and behavioral limitations are barriers to the social integration of patients. The PWS also has repercussions on well-being and quality of life of the family. Thus, the present project focuses on the characteristics of emotional control related to the anger of children with PWS and its behavioral, cognitive and social implications. In addition, at a therapeutic level, our project assumes that transcutaneous electrical nerve stimulation (t-VNS), a non-invasive, safe and inexpensive method, could be effective in reducing the severity of behavioral disorders such as temper tantrums while improving cognitive performance and social communication in the PWS.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Questionnaires, t-VNS
- Keywords
- Emotion, Cognition
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnairesBehavioural
Evaluation of behavioral disorders of children with PWS by questionnaires
t-VNSDevice
Evaluation of the influence of 6 month-stimulation by t-VNS on emotional, cognitive, and behavioral/clinical characteristics of 12 children with PWS on 30 PWS patients included.
| Study group | Role | What they receive |
|---|---|---|
| children with PWS | Experimental | Evaluation of cognitive abilities of children with PWS by several cognitive tasks and neuropsychological tests. Receives: Behavioral: Questionnaires; Device: t-VNS |
| non affected children | Other | Evaluation of cognitive abilities of children with PWS compared to a non-pathologic population of children by several cognitive tasks and neuropsychological tests Receives: Behavioral: Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
assessment of the level of emotional control
composite score of emotional control level of children with PWS composed of scores of emotional lability
Measured: day 0
Secondary outcomes (5)
Evaluation of behavioral disorders of children with PWS by CBCL (Child Behavior Checklist) scale
CBCL (Child Behavior CheckList) scale: is a questionnaire comprising 1) an adaptive functioning scale (activities, social and school spheres), 2) a syndrome scale (social withdrawal, anxiety / depression, somatic complaints, social problems, thinking problems, attention problems, behavior problems and aggressiveness; total of internalized problems and total of externalized problems), 3) a DSM-oriented scale (Lengua et al., 2001; anxiety, attention problems / hyperactivity, behavior problems, depression, opposition / mistrust, social problems / immaturity, somatization). (duration = 20 ') - there is no minimum and maximum values as this is an evaluation of behavioral disorders
Measured: month 6
Evaluation of behavioral disorders of children with PWS by Hyperphagia Questionnaire
HQ-CT (Hyperphagia Questionnaire) scale: The HQ-CT (Hyperphagia Questionnaire for Use in Prader-Willi Syndrome Clinical Trials, Fehnel et al., 2015, appendix 10) is a scale derived from the Dykens hyperphagia questionnaire (Dykens et al., 2007). The specificity of the Dykens questionnaire makes it a favored tool in the international literature in the evaluation of the eating behavior of patients with PWS (Crain, 2010). The HQ-CT was created specifically to assess the eating behavior of these patients in clinical trials. It consists of 9 items for which a person taking care of the patient (family or caregiver) indicates the frequency and severity of the behavior described on a 5-point Likert scale (with 1 = absence of the behavior and 5 = very frequent behavior or severe) (⍺ = .91). (duration = 5 ') - so 1 is the minimum value and 5 the maximum value and higher scores mean a worse outcome
Measured: month 6
Evaluation of cognitive abilities of children with PWS
emotional language task
Measured: 2 days
Test of evaluation of cognitive abilities
multimodal emotional recognition test MERT
Measured: 2 days
Evaluation of cognitive abilities
WISC-V
Measured: 2 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2020
- Study start7 Sep 2020
- Primary completion7 Dec 2020
- Study completion27 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- The study consists of 2 parts. The first part compares 30 children with PWS compared to 30 control children without PWS. The second part will be based on 12 children with PWS who have undergone part 1.
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maithé TAUBER, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04526379
- Sponsor's protocol ID
- RC31/18/0335
- Acronym
- PRACOM1
- Status last verified
- Aug 2023
- First submitted
- 3 Mar 2020
- Last updated
- 23 Aug 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Communication in Prader-Willi Syndrome: Study of Emotional Control Related to Behavioral Disorders, Their Daily Repercussions and Examination of an Innovative Therapy: Transcutaneous Electrical Nerve Stimulation of the Vagus Nerve - PRACOM1
- ClinicalTrials.gov ID
- NCT04526379
- Acronym / sponsor ID
- PRACOM1 / RC31/18/0335
- Conditions
- Prader-Willi Syndrome
- Keywords
- Emotion, Cognition
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 7 Sep 2020 (actual)
- Primary completion
- 7 Dec 2020 (actual)
- Study completion
- 27 Apr 2022 (actual)
- First posted
- 25 Aug 2020 (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.
The present project project is divided in two parts. The primary aim of the part 1 of this study is to evaluate emotional control abilities of children with Prader-Willi syndrome (PWS) aged from 9 to 15 years and to study repercussions of this supposed lack of abilities on cognitive capacities and behavioral troubles. The study also evaluate influence of the emotional symptomatology of patients on quality of parents' life and on the care of parents and scholar/institutional caregivers. In the second part of this study, the study evaluate the feasibility and the tolerance to a non-invasive device supposed to reduce emotional symptoms in this disease.
Detailed description
Prader-Willi Syndrome (PWS) is a rare genetic disorder involving a variety of clinical, behavioral and cognitive symptoms. 83 to 97% of patients have episodes of temper tantrum, associated with an important emotional lability. These different cognitive and behavioral limitations are barriers to the social integration of patients. The PWS also has repercussions on well-being and quality of life of the family. Thus, the present project focuses on the characteristics of emotional control related to the anger of children with PWS and its behavioral, cognitive and social implications. In addition, at a therapeutic level, our project assumes that transcutaneous electrical nerve stimulation (t-VNS), a non-invasive, safe and inexpensive method, could be effective in reducing the severity of behavioral disorders such as temper tantrums while improving cognitive performance and social communication in the PWS.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study consists of 2 parts. The first part compares 30 children with PWS compared to 30 control children without PWS. The second part will be based on 12 children with PWS who have undergone part 1.
| Arm | Type | Receives |
|---|---|---|
| children with PWS | Experimental | Behavioral: Questionnaires, Device: t-VNSEvaluation of cognitive abilities of children with PWS by several cognitive tasks and neuropsychological tests. |
| non affected children | Other | Behavioral: QuestionnairesEvaluation of cognitive abilities of children with PWS compared to a non-pathologic population of children by several cognitive tasks and neuropsychological tests |
Who may qualify
- Ages
- 9 to 15 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age from 9 to 15 years old
- Prader-Willi syndrome with identified genotype.
- No psychiatric disorder neither PWS.
- Severe global symptomatology attested by a CGI-S score
Key exclusion criteria (5 listed)
- Identified psychiatric or behavioral disorders
- Severe visual or hearing impairment.
- Sleep apnea syndrome treated with non-invasive ventilation,
- Epileptic seizures
- Cardiac disorders.
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 |
|---|---|---|
| QuestionnairesEvaluation of behavioral disorders of children with PWS by questionnaires | Behavioural | children with PWS, non affected children |
| t-VNSEvaluation of the influence of 6 month-stimulation by t-VNS on emotional, cognitive, and behavioral/clinical characteristics of 12 children with PWS on 30 PWS patients included. | Device | children with PWS |
Primary outcome
assessment of the level of emotional control
Time frame: day 0
composite score of emotional control level of children with PWS composed of scores of emotional lability
5 secondary outcomes
Evaluation of behavioral disorders of children with PWS by CBCL (Child Behavior Checklist) scale
Time frame: month 6
Evaluation of behavioral disorders of children with PWS by Hyperphagia Questionnaire
Time frame: month 6
Evaluation of cognitive abilities of children with PWS
Time frame: 2 days
Test of evaluation of cognitive abilities
Time frame: 2 days
Evaluation of cognitive abilities
Time frame: 2 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 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.
Maithé TAUBER, MD principal investigator
University Hospital, Toulouse
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Questionnaires and t-VNS in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 1 site in France.
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 4 inclusion and 5 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: finished, as of the sponsor's last update on 23 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
assessment of the level of emotional control (time frame: day 0). 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 23 Aug 2023.