Treatment of NF1-related Plexiform Neurofibroma With Trametinib
Status:
Recruiting
Trial end date:
2022-07-01
Target enrollment:
Participant gender:
Summary
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.