Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 (NF1-QDV)
|ClinicalTrials.gov Identifier: NCT01851135|
Recruitment Status : Completed
First Posted : May 10, 2013
Last Update Posted : July 6, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis Type 1||Other: Neuropsychological examination and quality of life measures||Not Applicable|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||286 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1|
|Study Start Date :||May 2013|
|Actual Primary Completion Date :||April 2016|
|Actual Study Completion Date :||April 2016|
|Experimental: Patients with NF1||Other: Neuropsychological examination and quality of life measures|
|Healthy controls||Other: Neuropsychological examination and quality of life measures|
- 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.)
- 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 ]The neuropsychological tests used in this study evaluate different domains: language, visuoperceptive and visuomotor abilities, memory, attention, executive function and intelligence skills.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01851135
|Assistance Publique - Hôpitaux de Paris (Hôpital Henri Mondor)|
|Créteil, France, 94010|
|Centre Hospitalier Universitaire de Nantes|
|Nantes, France, 44093|
|Principal Investigator:||Sébastien Barbarot, MD||Nantes University Hospital|