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NCT03741101: plexifpc

Treatment of NF1-related Plexiform Neurofibroma With Trametinib

Active, enrolled Phase 2 Last updated 11 April 2024
What this trial tests

Phase 2 trial testing Trametinib in Neurofibromatosis 1 in 15 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
10 June 2019
Primary endpoint
15 July 2024
15 December 2024

Quick facts

Lead sponsorRegion Skane
PhasePhase 2
StatusActive, enrolled
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment15
Start date10 June 2019
Primary completion15 July 2024
Estimated completion15 December 2024
Sites1 location across Sweden

Drugs / interventions tested

Conditions studied

Sponsor

Region Skane — full company profile →

Who can join

Adults 1 to 17, any sex, with Neurofibromatosis 1 or Child. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Current Understanding of Neurofibromatosis Type 1, 2, and Schwannomatosis.
    Tamura R. · · 2021 · cited 153× · PMID 34072574 · DOI 10.3390/ijms22115850
  2. Management of neurofibromatosis type 1-associated plexiform neurofibromas.
    Fisher MJ, Blakeley JO, Weiss BD, Dombi E, et al · · 2022 · cited 104× · PMID 35657359 · DOI 10.1093/neuonc/noac146
  3. Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review.
    Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, et al · · 2021 · cited 35× · PMID 34388689 · DOI 10.1016/j.esmoop.2021.100223
  4. Neurofibromatosis in the Era of Precision Medicine: Development of MEK Inhibitors and Recent Successes with Selumetinib.
    Galvin R, Galvin R, Watson AL, Largaespada DA, et al · · 2021 · cited 19× · PMID 33721151 · DOI 10.1007/s11912-021-01032-y
  5. Understanding the Biological Activities of Vitamin D in Type 1 Neurofibromatosis: New Insights into Disease Pathogenesis and Therapeutic Design.
    Riccardi C, Perrone L, Napolitano F, Sampaolo S, et al · · 2020 · cited 15× · PMID 33066259 · DOI 10.3390/cancers12102965
  6. Current state of spinal nerve sheath tumor management and future advances.
    Gui C, Canthiya L, Zadeh G, Suppiah S. · · 2024 · cited 5× · PMID 39430389 · DOI 10.1093/noajnl/vdae067
  7. Neurofibromatosis: Molecular Pathogenesis and Natural Compounds as Potential Treatments.
    Amaravathi A, Oblinger JL, Welling DB, Kinghorn AD, et al · · 2021 · cited 5× · PMID 34604034 · DOI 10.3389/fonc.2021.698192
  8. The Contribution of Oxidative Stress to <i>NF1</i>-Altered Tumors.
    Kuhn E, Natacci F, Corbo M, Pisani L, et al · · 2023 · cited 4× · PMID 37627552 · DOI 10.3390/antiox12081557

Verify or expand the search:

Other trials of Trametinib

Trials testing the same drug.

Other recruiting trials for Neurofibromatosis 1

Currently open trials in the same condition.

Other Region Skane trials

Trials by the same sponsor.

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