a study on Cancer, General
This is a 3-part (Part A, Part B, Part C), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors. Part A is a repeat dose, dose escalation monotherapy study that will identify the recommended phase II dose (RP2D) on the continuous dosing schedule using a 3 + 3 dose-escalation procedure. Part B will evaluate the preliminary activity of trametinib monotherapy in 4 disease-specific cohorts of subjects. Each cohort will enroll at least 10 response-evaluable subjects (evaluable for response is defined as a subject with a pre-dose and at least 1 post-dose disease assessment or clinical assessment of progression of disease). Part C is will be a 3+3 study design to determine the safety, tolerability and preliminary activity of the RP2D of trametinib in combination with a limited dose escalation of dabrafenib. Part C will enroll up to 18 subjects. . The overall goal of this trial is to efficiently establish safe, pharmacologically relevant dose of trametinib in infants, children and adolescents and determine preliminary activity of trametinib monotherapy in selected recurrent, refractory or unresectable childhood tumors.
An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Children and Adolescents With Cancers Harboring V600 Mutations
Cancer v600-mutation neuroblastoma Trametinib pediatrics low grade glioma plexiform neurofibromas Dabrafenib
Open to people ages 1 month to 17 years
Adequate bone marrow function defined as absolute neutrophil count (ANC)
=1000/microliter, hemoglobin>=8.0 gram per deciliter (g/dL) (may receive red blood cell transfusions), platelets>=75,000/ microliter (transfusion independent, defined as not receiving platelet transfusions within a 7 day period prior to enrollment).
NOTE: Subjects previously treated for these conditions that have had stable CNS disease (verified with consecutive imaging studies) for>3 months, are asymptomatic and are not currently taking corticosteroids, or are on stable dose or decreasing of corticosteroids for at least 7 days prior to enrolment are permitted.
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