This phase II trial studies the effect of larotrectinib in treating patients with NTRK gene amplification positive solid tumors that have spread to nearby tissues or lymph nodes (locally advanced) or other places in the body (metastatic). Larotrectinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
PRIMARY OBJECTIVE: I. To determine overall response rate (ORR) to larotrectinib sulfate (larotrectinib) in patients with advanced solid tumors harboring NTRK amplification and pan-TRK expression by immunohistochemistry (IHC), calculated as the proportion of subjects with confirmed complete (CR) or partial response (PR) as best response and as assessed by Response Evaluation Criteria in Solid Tumors (RECIST) version (v) 1.1 and Response Assessment in Neuro-Oncology (RANO) criteria in primary central nervous system (CNS) tumor. SECONDARY OBJECTIVES: I. To evaluate the duration of response (DOR) in subjects with CR or PR as best response. II. To estimate the proportion of subjects with any tumor regression as best response. III. To evaluate the growth modulation index (GMI) following initiation of larotrectinib. IV. To evaluate overall (OS) and progression-free survival (PFS) following initiation of larotrectinib. V. To evaluate the clinical benefit rate (CBR) based on the proportion of subjects with best response of CR, PR, or stable disease lasting \>= 16 weeks following initiation of larotrectinib safety. VI. To assess the safety profile and tolerability of larotrectinib. EXPLORATORY OBJECTIVES: I. To characterize NTRK1, NTRK2, and NTRK3 amplification by next-generation sequencing of tumor biopsies. II. To characterize TRKA, TRKB, and TRKC signaling in fresh pre-treatment tumor biopsies, with the aim of elucidating TRK biology and modifiers of response to larotrectinib. III. To characterize concurrently activated oncogenic pathways in fresh pre-treatment tumor biopsies, with the aim of elucidating TRK biology and modifiers of response to larotrectinib. OUTLINE: Patients receive larotrectinib sulfate orally (PO) twice daily (BID) on days 1-28. Cycles repeat every 28 days in the absence of unacceptable toxicity. Patients who experience disease progression and are deriving clinical benefit from larotrectinib may continue treatment per physician discretion. After completion of study treatment, patients are followed up at 4 weeks, and then every 3 months for 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Given PO
M D Anderson Cancer Center
Houston, Texas, United States
RECRUITINGOverall response
Will be scored by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.
Time frame: Up to 2 years post-treatment
Best overall response of confirmed complete response or partial response
Will be determined using RECIST version 1.1 or Response Assessment in Neuro-Oncology criteria, as appropriate to tumor type. A bias-correcting estimate of the best overall response rate and its one-sided 90% confidence interval will be calculated using an exact inference method that appropriately accounts for Bayesian optimal phase II design.
Time frame: Up to 2 years post-treatment
Duration of response
Will be calculated for subjects who achieve confirmed complete response or partial response, and Kaplan-Meier estimates, mean and median duration, and two-sided 95% confidence intervals will be generated.
Time frame: From the date complete response or partial response is first noted (whichever response is recorded first), to the date that recurrence or progressive disease is first documented or to date of death, assessed up to 2 years
Growth modulation index
Will be defined as the ratio of time to progression (TTP) with nth line of therapy (TTPn) to the most recent prior line of therapy and summarized with mean, median, standard error, and two-sided 95% confidence interval.
Time frame: Up to 2 years post-treatment
Progression-free survival
Kaplan-Meier estimates, mean and median duration, and two-sided 95% confidence intervals will be generated. In patients who have received prior therapy, corresponding Kaplan-Meier plots, mean and median duration with 95% confidence intervals will be used to compare the duration of progression-free survival following initiation of larotrectinib to that following the line of therapy immediately preceding larotrectinib.
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Purpose
TREATMENT
Masking
NONE
Enrollment
13
Time frame: From initiation of larotrectinib to disease progression or death due to any cause, whichever occurs first, assessed up to 2 years
Overall survival
Kaplan-Meier estimates, mean and median duration, and two-sided 95% confidence intervals will be generated.
Time frame: From initiation of treatment to death from any cause, assessed up to 2 years
Clinical benefit rate
Will be defined as the proportion of subjects with best overall response of complete response, partial response, or stable disease lasting 16 or more weeks following initiation of treatment and summarized in percentages with two-sided 95% confidence intervals.
Time frame: Up to 2 years post-treatment
Incidence of adverse events
Safety will be assessed by clinical review of all relevant parameters including adverse events, serious adverse events, laboratory values, vital signs, and electrocardiogram results. Overall safety profiles will be provided along with tumor-specific profiles. Treatment-emergent adverse events, defined as adverse events that start on or after the first administration of study drug, will be summarized based on the number and percentage of subjects experiencing the event, as defined by Medical Dictionary for Regulatory Activities system organ class and preferred term. In the event a subject experiences repeat episodes of the same adverse event, then the event with the highest severity grade and strongest causal relationship to study drug will be used for purposes of incidence tabulations.
Time frame: Up to 4 weeks (after the final dose of the last cycle of treatment (between 21 and 35 days)
Change in clinical safety laboratory values and vital signs
Waterfall plots will be used to depict graphically for individual subjects the maximum percentage decrease from baseline in the sum of the longest diameters of target lesions. Similarly, spider and swimmer plots will be used to display the change in tumor burden over time for individual subjects and the occurrence of clinical outcomes of interest (e.g., tumor response, disease progression, treatment discontinuation, death).
Time frame: Baseline up to 2 years post-treatment