Treatment of NF1-related Plexiform Neurofibroma With Trametinib (plexifpc)
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|ClinicalTrials.gov Identifier: NCT03741101|
Recruitment Status : Recruiting
First Posted : November 14, 2018
Last Update Posted : September 11, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis 1 Child Neurofibroma, Plexiform||Drug: Trametinib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||June 10, 2019|
|Estimated Primary Completion Date :||July 2022|
|Estimated Study Completion Date :||July 2022|
Experimental: single arm study
children treated with trametinib
Other Name: mekinist
- Remission of tumor volume ≥20% [ Time Frame: 0 - 30 months. ]Final and primary analysis of primary outcome measure - of pooled data at 30 months (end of study) with volumetric mri of tumor volume versus volume at enrolment.
- Remission of tumor volume ≥20% [ Time Frame: 0 - 18 months ]Interim analysis of pooled data at 18 months with volumetric mri of tumor volume versus volume at enrolment. This is an interim analysis of primary outcome and not primary analysis of primary outcome".
- Reversal of NF1-related PN elicited - VAS scale pain [ Time Frame: 0 - 30 months. ]Evaluated with monthly VAS pain scale from 8 years at enrolment. Monthly. 0-10 point scale. Descriptive. Analysis of pooled data after month 30.
- Reversal of NF1-related PN elicited pain - Faces Pain Scale [ Time Frame: 0 - 30 months. ]Evaluated with monthly Faces Pain Scale from under 8 years at enrolment. Monthly. 0-10 scale. Descriptive. Analysis of pooled data after month 30.
- Cognitive performance. Exploratory. WISC V. [ Time Frame: 0-18 months. ]Change of full scale IQ or primary indexes of WISC V. Pooled data after 18 months versus before/at enrolment; with p-value <0.05.
- Cognitive performance. Exploratory. NEPSYII. [ Time Frame: 0-18 months. ]Change of Learning and Memory functions and visuospatial functions, pooled data after 18 months versus before/at enrolment (selected test from NEPSYII,; with p-value <0.05.
- Cognitive performance. Exploratory. CPT3. [ Time Frame: 0-18 months. ]Change in attention, pooled data after 18 months versus before/at enrolment (CPT-3); with p-value <0.05.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03741101
|Contact: Björn Sigurdsson, MD, PIfirstname.lastname@example.org|
|Contact: Erik A Eklund, MD, PhDemail@example.com|
|Principal Investigator:||Björn Sigurdsson||Skane University Hospital|