Neurofibromatosis Type 1 Clinical Trial
Official title:
The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial (NF1-EXCEL)
The purpose of this study is to determine whether lamotrigine can improve cognitive and neurophysiological deficits in adolescents with Neurofibromatosis type 1.
Cognitive deficits in the autosomal dominant disorder Neurofibromatosis type 1 (NF1) typically consist of a lower than average IQ, impaired visual-spatial learning, attention problems and impaired executive functioning. These deficits have a substantial influence on the daily life of pediatric and adolescent individuals with NF1. One of the key underlying mechanisms of these deficits is an increased gamma-aminobutyric acid (GABA)-ergic inhibition and a subsequent decrease in synaptic plasticity. The ENCORE laboratory has recently shown that loss of the NF1-gene is associated with attenuated function of the hyperpolarization-activated cyclic nucleotide-gated channel 1 (HCN1). These channels, enriched in membranes of inhibitory interneurons, play an important role in the pathophysiology underlying the cognitive deficits in NF1. Lamotrigine, an HCN-agonist, restored function of HCN1, together with the electrophysiological and visual-spatial learning deficits in Nf1-mice. Thus, lamotrigine is a novel candidate drug for treating cognitive deficits associated with NF1. ;
| 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 | |
| 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 |
NCT01851135 -
Neuropsychological Impairment and Quality of Life 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 |