Treatment of NF1-related Plexiform Neurofibroma With Trametinib

Sponsor
Region Skane
Study ID
NCT03741101
Phase
PHASE2
Status
Unknown

Conditions

  • Child
  • Neurofibroma, Plexiform
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
1 Year - 17 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Jun 10, 2019
Status verified
Apr 2024
Primary completion
Jul 15, 2024
Completion
Dec 15, 2024

Study Design

Enrollment
15 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: single arm study
    children treated with trametinib

Primary Outcome Measure

Remission of tumor volume ≥20% [ Time Frame: 0 - 30 months. ]

Related Studies