Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 2 - Dovitinib for Patients With Tumor Pathway Activations Inhibited by Dovitinib Including Tumors With Mutations or Translocations of FGFR, PDGFR, VEGF, cKIT, FLT3, CSFR1, Trk and RET
This is a phase II, open label study to determine the efficacy and safety of treatment with
dovitinib (TKI258) in patients with a diagnosis of select solid tumors or hematological
malignancies that have been pre-identified (prior to study consent) to have mutations,
translocations or pathway activations inhibited by dovitinib and whose disease has
progressed on or after standard treatment.
Genomic profiling is becoming more accessible to patients and their physicians. As such,
more patients have been identified with potentially-actionable mutations, translocations or
pathway-activations but do not have access to targeted drug treatment. This is a
signal-seeking study to match patients with tumor pathway activations inhibited by dovitinib
to the RTK inhibitor dovitinib. Pre-identification of mutations, translocations, or tumor
pathway activations inhibited by dovitinib will be performed locally at a CLIA certified
laboratory prior to 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 dovitinib single-agent.
The patient may not receive any additional anti-cancer therapy during treatment with
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 (by RECIST 1.1 or appropriate hematological response assessment 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 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)
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Clinical benefit associated with dovitinib treatment
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.
United States: Food and Drug Administration
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