NCT02122913 · CITED IN SOURCE DOCUMENTS

A Study to Test the Safety of the Investigational Drug Larotrectinib in Adults That May Treat Cancer

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase: PHASE1
Status at capture: COMPLETED
Registry last update: 2026-02-05

This research study is done to test the safety of the drug larotrectinib in adult cancer patients. The drug may be used to treat cancer with a change in a particular gene (NTRK1, NTRK2 or NTRK3), because it blocks the action of these genes in cancer cells. The study also investigates how the drug is absorbed and processed in the human body. This is the first study to test larotrectinib in humans with cancer, for whom no other effective therapy exists.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part

eligibility Criteria: Inclusion Criteria: * Adult patients with a locally advanced or metastatic solid tumor that has progressed or was nonresponsive to available therapies, are unfit for standard chemotherapy or for which no standard or available curative therapy exists * Proof of a malignancy harboring a NTRK fusion * Eastern Cooperative Oncology Group (ECOG) score of 0, 1 or 2 and a life expectancy of at least 3 months * Adequate hematologic, hepatic, and renal function Exclusion Criteria: * Patients with unstable primary central-nervous-system tumors or metastasis, exceptions possible * Clinically significant active cardiovascular disease or history of myocardial infarction * Active uncontrolled systemic bacterial, viral, or fungal infection * Current treatment with a strong CYP3A4 inhibitor or inducer * Pregnancy or lactation
healthy Volunteers: false
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
Adult patients with solid tumors receiving 50 mg of BAY2757556 once daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 1
type
EXPERIMENTAL
description
Adult patients with solid tumors receiving 100 mg of BAY2757556 once daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 2
type
EXPERIMENTAL
description
Adult patients with solid tumors receiving 100 mg of BAY2757556 twice daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 3
type
EXPERIMENTAL
description
Adult patients with solid tumors receiving 200 mg of BAY2757556 once daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 4
type
EXPERIMENTAL
description
Adult patients with solid tumors receiving 150 mg of BAY2757556 twice daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 5
type
EXPERIMENTAL
description
Adult patients with solid tumors receiving 200 mg of BAY2757556 twice daily (dose escalation cohort).
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Dose 6
type
EXPERIMENTAL
description
Adults patients with solid tumors and neurotrophic tyrosine kinase (NTRK) genes or proteins of types 1 - 3 (dose expansion cohort). Patients receive either the recommended or maximum tolerated dose of BAY2757556 as determined in the dose escalation part.
intervention Names
Drug: Larotrectinib (Vitrakvi, BAY2757556)
label
Tumor patients_Expansion
type
EXPERIMENTAL

interventions

arm Group Labels
Tumor patients_Dose 1
Tumor patients_Dose 2
Tumor patients_Dose 3
Tumor patients_Dose 4
Tumor patients_Dose 5
Tumor patients_Dose 6
Tumor patients_Expansion
description
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
name
Larotrectinib (Vitrakvi, BAY2757556)
other Names
LOXO-101
type
DRUG

Study design

allocation

RANDOMIZED

intervention Model

SEQUENTIAL

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 75
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

measure
Number of participants with adverse events
time Frame
25 months
description
The severity of adverse events will be assesssed according to the NCI CTCAE version 4.03.
measure
Severity of adverse events
time Frame
25 months
measure
Maximum tolerated dose (MTD)
time Frame
25 months
measure
Recommended dose for dose expansion
time Frame
25 months

secondary Outcomes

measure
Maximum concentration of larotrectinib in plasma (Cmax)
time Frame
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
measure
Time to maximum concentration of larotrectinib in plasma (Tmax)
time Frame
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
measure
Half-life of larotrectinib in plasma (t1/2)
time Frame
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
measure
Area under the concentration versus time curve of larotrectinib in plasma (AUC)
time Frame
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
description
Assessed using the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or Response Assessment in Neuro-Oncology (RANO) as appropriate
measure
Overall Response Rate (ORR)
time Frame
Up to 60 months
measure
Duration of Response (DOR)
time Frame
Up to 60 months

Full study description

brief Summary: This research study is done to test the safety of the drug larotrectinib in adult cancer patients. The drug may be used to treat cancer with a change in a particular gene (NTRK1, NTRK2 or NTRK3), because it blocks the action of these genes in cancer cells. The study also investigates how the drug is absorbed and processed in the human body. This is the first study to test larotrectinib in humans with cancer, for whom no other effective therapy exists.
detailed Description: The trial will be conducted in 2 parts: an initial dose escalation phase of larotrectinib in subjects with advanced solid tumors will be followed by an expansion phase in subjects with solid tumors having a NTRK fusion. The objectives of the study are to determine the safety, pharmacokinetic profile, recommended dose and efficacy of orally administered larotrectinib in patients with NTRK fusions.

Source references

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