Child Clinical Trial
Official title:
Treatment of NF1-related Plexiform Neurofibroma With Trametinib; a Single Arm, Open-label Trial With the Goals of Volumetric Partial Remission and Pain Relief
This trial, Treatment of NF1-related plexiform neurofibroma with trametinib; a single arm,open-label study with the goals of volumetric partial remission and pain relief (EudraCT 2018-001846-32, Sponsor protocol number BUS2018-1, related Novartis reference number CTMT212ASE01T) is a pediatric clinical trial that investigates the potential use of the drug trametinib (Mekinist®) as treatment for symptomatic or likely to become symptomatic NF1-related plexiform neurofibromas (PN) in children between 1 year and 17 year and 11 months of age. Trametinib is orally administered qd at 0.025 mg/kg up to a maximum of 2 mg from six years of age and 0.032mg/kilo up to 5 years of age, provided either as tablets or as oral solution. It is manufactured and distributed by Novartis under the trade name Mekinist®. The primary endpoint is remission of tumor volume ≥20%, evaluated using volumetric MRI at 18 and 30 months of treatment. The secondary endpoint is reversal of pain from NF1-related PN, evaluated monthly with agespecific pain scales; VAS scale (from 8 years) or Faces Pain Scale (from 3 to 8 years). As an exploratory measure, the potential effects of the treatment on the cognitive function will be assessed using well-established tests such as WISC-V (age range 6:0 - 16:11), NEPSY-II (age range 3:0-16:11), and CPT-3 (age range 8:0 - adult). Cognitive dysfunction is well described in patients with NF1, and the MAPK/ERK-pathway has been indicated to be involved in cognition.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT06320626 -
Pharmacokinetic-guided Dosing of Emicizumab
|
Phase 4 | |
| Completed |
NCT04011852 -
The Role of Chest Electrical Impedance Tomography in the Pediatric Ventilator Weaning
|
||
| Completed |
NCT03497663 -
VIA Family - Family Based Early Intervention Versus Treatment as Usual
|
N/A | |
| Recruiting |
NCT05016271 -
Health Benefits of Air Purifiers in Primary School Students
|
N/A | |
| Completed |
NCT02551978 -
Kids Obesity Prevention Program - Study (KOP)
|
N/A | |
| Completed |
NCT02240368 -
Performance Evaluation of the Depth of Anesthesia Monitors in Pediatric Surgery
|
||
| Completed |
NCT03946137 -
Chest Therapy and Postural Education in Children With Cerebral Palsy
|
N/A | |
| Completed |
NCT01918020 -
Impact Study of Minnesota SNAP-Ed Health and Nutrition Education Programs
|
N/A | |
| Completed |
NCT01267097 -
The PAC Study: Parents as Agents of Change in Pediatric Weight Management
|
N/A | |
| Recruiting |
NCT04022460 -
Using Personal Mobile Technology to Identify Obstructive Sleep Apnea in Children With Down Syndrome (UPLOAD)
|
||
| Completed |
NCT05538039 -
Reducing Anxiety of Children and Their Parents in the Pre-elective Surgery Process
|
N/A | |
| Completed |
NCT03156465 -
Iowa Cochlear Implant Clinical Research Center Hybrid L24 and Standard Cochlear Implants in Profoundly Deaf Infants
|
N/A | |
| Not yet recruiting |
NCT05914324 -
Outpatient Pediatric Pulse Oximeters in Africa
|
N/A | |
| Not yet recruiting |
NCT04827108 -
Psychometric Properties of the Chinese Version of PeNAT
|
||
| Recruiting |
NCT04888195 -
Symptom Clusters in Hong Kong Chinese Children With Blood Cancer
|
||
| Not yet recruiting |
NCT04853303 -
VR to Improve CINV, Sleep and Pain Among Children With Cancer in HK
|
N/A | |
| Completed |
NCT05374902 -
Efficacy of Animation, Buzzy, and Multiple Interventions on Pain in Children
|
N/A | |
| Completed |
NCT04665453 -
Dexmedetomidine and Melatonin for Sleep Induction for EEG in Children
|
N/A | |
| Completed |
NCT04082247 -
Healthy Children 2021 Study in Childcare Centers
|
N/A | |
| Completed |
NCT02858531 -
Predictive Tracking of Patient Flow in the Emergency Services During the Virus Winter Epidemics
|