Neurofibromatosis Type 1 Clinical Trial
Official title:
Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1
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.
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.
;
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT03190915 -
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
|
Phase 2 | |
| Completed |
NCT03326388 -
Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours
|
Phase 1/Phase 2 | |
| Completed |
NCT00026780 -
Eligibility Screening for a NCI Pediatric Oncology Branch Research Study
|
||
| Active, not recruiting |
NCT01362803 -
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT01218139 -
Analysis of Peripheral Nerve Sheath Tumors (PNSTs) in Neurofibromatosis Type 1 (NF1) Patients
|
N/A | |
| Recruiting |
NCT05149469 -
Molecular Aspects of Preimplantation Genetic Diagnosis for NF1
|
||
| Not yet recruiting |
NCT02505412 -
Subtle Myocardial Deformation Abnormalities in Asymptomatic Nf-1 Patients
|
N/A | |
| Terminated |
NCT02256124 -
Effect of Lamotrigine on Cognition in NF1
|
Phase 2/Phase 3 | |
| Completed |
NCT01707836 -
Neurofibromatosis Type 1 Brain Tumor Genetic Risk
|
N/A | |
| Active, not recruiting |
NCT01218152 -
Microarray CGH Analysis of Circulating Tumoral Plasma DNA in NF1 Patients With MPNSTs
|
N/A | |
| Recruiting |
NCT03975829 -
Pediatric Long-Term Follow-up and Rollover Study
|
Phase 4 | |
| Recruiting |
NCT05186870 -
Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest
|
||
| Active, not recruiting |
NCT03231306 -
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas
|
Phase 2 | |
| Withdrawn |
NCT03332030 -
Stem Cells in NF1 Patients With Tumors of the Central Nervous System
|
||
| Recruiting |
NCT02964884 -
Interventions for Reading Disabilities in NF1
|
Phase 2 | |
| Recruiting |
NCT05388370 -
PASS of Paediatric Patients Initiating Selumetinib
|
||
| Recruiting |
NCT02777775 -
Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.
|
||
| Completed |
NCT02944032 -
Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
|
N/A | |
| Completed |
NCT01410006 -
Neurofibromatosis Type 1 Patient Registry
|
N/A | |
| Active, not recruiting |
NCT00326872 -
AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine
|
Phase 2 |