MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1
Status:
Completed
Trial end date:
2018-08-01
Target enrollment:
Participant gender:
Summary
This phase II open label study will evaluate adolescents (≥ 16 years of age) and adults with
neurofibromatosis type-1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor
PD-0325901. The primary aim of the study will be to assess quantitative radiographic response
in a target lesion. Subjects will receive PD-0325901 by mouth on a bid dosing schedule of 2
mg/m2/dose with a maximum dose of 4 mg bid. Each course is 4 weeks duration, and subjects
will receive drug on a 3 week on/1 week off schedule. Subjects may receive additional courses
beyond course 8 only if there is at least 15% reduction in volume of the target tumor.
Subjects who have a 20% or greater reduction in target tumor volume at the end of 12 courses
can continue on therapy for up to an additional year (maximum of 24 total courses). However,
subjects who do not achieve at least 15% reduction in volume of the target tumor after 8
courses (~8 months) will be considered treatment failures and taken off study.
The Primary purpose of this protocol is to determine whether PD-0325901 results in objective
radiographic responses based on volumetric MRI measurements in adolescents and adults with
NF1 and growing or symptomatic inoperable PN.
There are several secondary aims of this protocol:
To evaluate the feasibility and toxicity of chronic PD-0325901 administration in this patient
population
To estimate the objective response rate of up to 2 non-target plexiform neurofibromas to
PD-0325901 by MRI
To characterize the pharmacokinetic profile of PD-0325901 when administered to this patient
population
To evaluate quality of life and pain during treatment with PD-0325901