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Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 3 - MEK162 for Patients With RAS/RAF/MEK Activated Tumors

Phase 2
18 Years
Not Enrolling
Solid Tumor and Hematologic Malignancies

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Trial Information

Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 3 - MEK162 for Patients With RAS/RAF/MEK Activated Tumors

This is a phase II, open label study to determine the efficacy and safety of treatment with
MEK162 in patients with a diagnosis of select solid tumors or hematological malignancies
that have been pre-identified (prior to study consent) to have activations of the
RAS/RAF/MEK pathway and whose disease has progressed on or after standard treatment.

Genomic profiling is becoming more accessible to patients and their physicians. This is a
signal-seeking study to match patients with mutations in RAF, RAS, NF1 or MEK to the
ATP-noncompetitive MEK 1/2 inhibitor, MEK162. Pre-identification of these mutations or
activations in the pathway will be performed locally at a CLIA certified laboratory prior to
screening for participation on the trial.

Once the patient has been identified, treating physicians who are qualified investigators
may contact Novartis to consider enrollment in this study. For the purpose of this study,
genomic profiling is not considered part of screening. Informed consent must be signed
before any screening activities take place. Once eligibility (screening criteria met) has
been confirmed by Novartis, the patient will initiate therapy with single agent MEK162. The
patient may not receive any additional anti-cancer therapy during treatment with MEK162.

Patients will continue to receive study treatment until disease progression (assessed by
RECIST 1.1 or appropriate hematologic response criteria), unacceptable toxicity, death or
discontinuation from study treatment for any other reason (e.g., withdrawal of consent,
start of a new anti-neoplastic therapy or at the discretion of the investigator), otherwise
known as End of Treatment. All patients who discontinue from study treatment due to disease
progression must have their progression clearly documented.

Disease assessment (per RECIST 1.1 or appropriate hematological response criteria) will be
performed every 8 weeks (±4 days) after first dose of study drug (Day 1 of every odd cycle),
until disease progression or end of treatment, whichever occurs first. The frequency of
disease assessment may be reduced to every 12 weeks for patients who have at least 4
post-baseline disease assessments and are clinically stable (except AML and MM patients).
Scans will be assessed locally by the investigator. After discontinuation of treatment,
patients, regardless of reason for treatment discontinuation, will be followed for safety
for 30 days after the last dose.

All patients will be followed for survival status every 3 months for 2 years after the last
patient has enrolled in the study regardless of treatment discontinuation reason (except if
consent is withdrawn or patient is lost to follow-up.)

Inclusion Criteria:

- Patient has a confirmed diagnosis of a select solid tumor (except for primary
diagnosis of pancreatic cancer, biliary cancer, colorectal cancer, low grade serous
ovarian cancer, melanoma) or hematologic malignancy (except for primary diagnosis of
chronic myelomonocytic leukemia).

- Patients must be pre-identified as having a tumor with a mutation in RAF, RAS, NF1 or
MEK at a CLIA certified laboratory

- Patient must have received at least one prior treatment for recurrent, metastatic and
/or locally advanced disease and for whom no standard therapy options are anticipated
to result in a durable remission.

- Patient must have progressive and measurable disease as per RECIST 1.1. or other
appropriate hematological guidelines.

- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

Exclusion Criteria:

- Patient has received prior treatment with MEK162.

- Patients with primary CNS tumor or CNS tumor involvement

- History of retinal degenerative disease

- History or current evidence of central serous retinopathy (CSR) or retinal vein
occlusion (RVO)

- Any ophthalmopathy visible at screening that would be considered a risk factor for
CSR or RVO by the ophthalmologist

- Patients who have neuromuscular disorders that are associated with elevated CK

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Clinical benefit rate associated with MEK162 treatment

Outcome Description:

Clinical benefit rate for patients with solid tumors will be assessed using RECIST 1.1 and will include responses of CR or PR or SD. For hematologic tumors other appropriate hematological response criteria will apply

Outcome Time Frame:

16 weeks

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

July 2013

Completion Date:

April 2017

Related Keywords:

  • Solid Tumor and Hematologic Malignancies
  • RAS, RAF, MEK, NF1, MEK162, signature, papillary thyroid, small intestine, bladder, esophagus, lung cancer, NSCLC, acute myelogenous leukemia, AML
  • Neoplasms
  • Hematologic Neoplasms