Neurofibromatosis Type 1NF1-QDVNCT01851135
Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1
A study testing Neuropsychological examination and quality of life measures in people with Neurofibromatosis Type 1, run by Nantes University Hospital at 2 sites in France.
Trial summary
The main objective of the study is to investigate the determinants of the quality of life in children and adults with Neurofibromatosis type 1 (NF1) and more particularly the specific impact of neuropsychological deficits. Read more about this study
- Who is it for?
- Ages 8 to 59 yearsHealthy volunteers can take part
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- Neuropsychological examination and quality of life measures
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 286 participants
- Where
- 2 sitesFrance
- Study dates
- May 2013to Apr 2016
- 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 8 to 59 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- NF1 diagnosis following National Institutes of Health criteria (1988)
- Age: 8-12 years for children, 18-59 years for adults
- Signed informed written consent (parent and patient for children, patient for adults)
- French speaking
- Resident in France
- Absence of NF1 diagnosis, learning disabilities, intellectual precocity
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- Age: 8-12 years for children, 18-59 years for adults
- Signed informed written consent (parent and healthy control for children, healthy control for adults)
- French speaking
- Resident in France
Who cannot join
- Uncorrectable hearing of visual impairment
- History of psychiatric illness
- Neuropsychological investigation in the last 6 months
- Insufficient language usage
- Any other known history of central nervous system pathology or neuropathological complications of NF1
Trial locations and contacts
2 sites in 1 country.
France2 sites
Assistance Publique - Hôpitaux de Paris (Hôpital Henri Mondor)
Créteil, 94010
Centre Hospitalier Universitaire de Nantes
Nantes, 44093
About this study
The main objective of the study is to investigate the determinants of the quality of life in children and adults with Neurofibromatosis type 1 (NF1) and more particularly the specific impact of neuropsychological deficits. In fact, cognitive impairment is currently considered as one of the most pervasive features of this genetic disorder but its relationship with the worsening of quality of life found in this population has not been directly investigated to date.
Secondary objectives of this study are (i) to compare neuropsychological and quality of life measures between patients and healthy controls matched by age, gender and education level, (ii) to contrast neuropsychological deficits incidence between patients and controls, and (iii) to differentiate NF1 children's self versus hetero-assessment of quality of life.
The main hypothesis of this study is that the neuropsychological impairment classically identified in this clinical population will be associated to the quality of life's worsening both in children and adults.
Read the detailed description
The study cohort will consist of 150 patients with NF1 (100 adults and 50 children) recruited from the Neurofibromatosis Clinic at the University Hospital in Nantes and Créteil (France). A sample of 150 healthy controls (100 adults and 50 children) will also be recruited from sport and leisure clubs to serve as a normally developing control group.
The protocol assessment includes a standard and thorough neuropsychological examination specific to children and adults, to investigate the different aspects of cognitive domains: language, visuoperceptive and visuomotor abilities, memory, attention, executive function and intelligence skills. Quality of life is measured by a questionnaire specifically adapted to children and adults.
Other factors linked to the NF1 disease (familial or sporadic form, severity and visibility) and to demographic characteristics (sex, age, education level) will be taken into account to study their respective impact on quality of life, as compared with neuropsychological measures.
- Conditions
- Neurofibromatosis Type 1
- Being tested
- Neuropsychological examination and quality of life measures
- Keywords
- Quality of life, Neuropsychological disorders, Neurofibromatosis type 1
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neuropsychological examination and quality of life measuresOther
| Study group | Role | What they receive |
|---|---|---|
| Patients with NF1 | Experimental | Not described Receives: Other: Neuropsychological examination and quality of life measures |
| Healthy controls | Other | Not described Receives: Other: Neuropsychological examination and quality of life measures |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life as measured by the questionnaires used in children and adults
Several domains in relation with quality of life (self perception, well-being, social life, emotions, etc.)
Measured: at day 0 for adults; at 4 weeks for children
Secondary outcomes (1)
Scores obtained from neuropsychological tests (composite measure)
The neuropsychological tests used in this study evaluate different domains: language, visuoperceptive and visuomotor abilities, memory, attention, executive function and intelligence skills.
Measured: at day 0 and 2 weeks for adults; at day 0, 2 weeks and 4 weeks for children
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2013 (estimated)
- Study startMay 2013
- Primary completionApr 2016
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Nantes University HospitalThe 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
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 286 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nantes University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Angers University, Association Neurofibromatoses et Recklinghaüsen
- Responsible party
- Sponsor
- Investigators
- Sébastien Barbarot, MD, Principal investigator, Nantes University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01851135
- Sponsor's protocol ID
- RC12_0130
- Acronym
- NF1-QDV
- Status last verified
- Jul 2016
- First submitted
- 24 Apr 2013
- Last updated
- 6 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT01851135
- Acronym / sponsor ID
- NF1-QDV / RC12_0130
- Conditions
- Neurofibromatosis Type 1
- Keywords
- Quality of life, Neuropsychological disorders, Neurofibromatosis type 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 286 participants (actual)
- Lead sponsor
- Nantes University HospitalUniversity, hospital or other
- Collaborators
- Angers University, Association Neurofibromatoses et Recklinghaüsen
- Study start
- May 2013
- Primary completion
- Apr 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 10 May 2013 (estimated)
- 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 main objective of the study is to investigate the determinants of the quality of life in children and adults with Neurofibromatosis type 1 (NF1) and more particularly the specific impact of neuropsychological deficits. In fact, cognitive impairment is currently considered as one of the most pervasive features of this genetic disorder but its relationship with the worsening of quality of life found in this population has not been directly investigated to date.
Secondary objectives of this study are (i) to compare neuropsychological and quality of life measures between patients and healthy controls matched by age, gender and education level, (ii) to contrast neuropsychological deficits incidence between patients and controls, and (iii) to differentiate NF1 children's self versus hetero-assessment of quality of life.
The main hypothesis of this study is that the neuropsychological impairment classically identified in this clinical population will be associated to the quality of life's worsening both in children and adults.
Detailed description
The study cohort will consist of 150 patients with NF1 (100 adults and 50 children) recruited from the Neurofibromatosis Clinic at the University Hospital in Nantes and Créteil (France). A sample of 150 healthy controls (100 adults and 50 children) will also be recruited from sport and leisure clubs to serve as a normally developing control group.
The protocol assessment includes a standard and thorough neuropsychological examination specific to children and adults, to investigate the different aspects of cognitive domains: language, visuoperceptive and visuomotor abilities, memory, attention, executive function and intelligence skills. Quality of life is measured by a questionnaire specifically adapted to children and adults.
Other factors linked to the NF1 disease (familial or sporadic form, severity and visibility) and to demographic characteristics (sex, age, education level) will be taken into account to study their respective impact on quality of life, as compared with neuropsychological measures.
Study design
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with NF1 | Experimental | Other: Neuropsychological examination and quality of life measures |
| Healthy controls | Other | Other: Neuropsychological examination and quality of life measures |
Who may qualify
- Ages
- 8 to 59 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- NF1 diagnosis following National Institutes of Health criteria (1988)
- Age: 8-12 years for children, 18-59 years for adults
- Signed informed written consent (parent and patient for children, patient for adults)
- French speaking
- Resident in France
Key exclusion criteria (5 listed)
- Uncorrectable hearing of visual impairment
- History of psychiatric illness
- Neuropsychological investigation in the last 6 months
- Insufficient language usage
- Any other known history of central nervous system pathology or neuropathological complications of NF1
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 |
|---|---|---|
| Neuropsychological examination and quality of life measures | Other | Healthy controls, Patients with NF1 |
Primary outcome
Quality of life as measured by the questionnaires used in children and adults
Time frame: at day 0 for adults; at 4 weeks for children
Several domains in relation with quality of life (self perception, well-being, social life, emotions, etc.)
1 secondary outcome
Scores obtained from neuropsychological tests (composite measure)
Time frame: at day 0 and 2 weeks for adults; at day 0, 2 weeks and 4 weeks for children
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 6 Jul 2016.
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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.
Sébastien Barbarot, MD principal investigator
Nantes University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Neuropsychological examination and quality of life measures in people with Neurofibromatosis Type 1, run by Nantes University Hospital at 2 sites 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 10 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 6 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Quality of life as measured by the questionnaires used in children and adults (time frame: at day 0 for adults; at 4 weeks for children). 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 6 Jul 2016.