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

February 3, 2026 updated by: Bayer

A Phase 1 Study of the Oral TRK Inhibitor Larotrectinib in Adult Patients With Solid Tumors

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

75

Phase

  • Phase 1

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • UCHealth Cancer Center - Anschutz Medical Campus - University of Colorado Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Mass General Cancer Center
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • UH Seidman Cancer Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • OHSU Hospital - Neurology
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Hospital of the University of Pennsylvania - Radiology
      • Pittsburgh, Pennsylvania, United States, 15219
        • UPMC Mercy - Oncology
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • SCRI Oncology Partners
    • Texas
      • Houston, Texas, United States, 77030
        • The University of Texas MD Anderson Cancer Center - Texas Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tumor patients_Dose 1
Adult patients with solid tumors receiving 50 mg of BAY2757556 once daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Dose 2
Adult patients with solid tumors receiving 100 mg of BAY2757556 once daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Dose 3
Adult patients with solid tumors receiving 100 mg of BAY2757556 twice daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Dose 4
Adult patients with solid tumors receiving 200 mg of BAY2757556 once daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Dose 5
Adult patients with solid tumors receiving 150 mg of BAY2757556 twice daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Dose 6
Adult patients with solid tumors receiving 200 mg of BAY2757556 twice daily (dose escalation cohort).
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101
Experimental: Tumor patients_Expansion

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.
Drug: Larotrectinib (Vitrakvi, BAY2757556)
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
Other Names:
  • LOXO-101

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
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
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
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
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
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
Predose and 0.25, 0.5, 1, 2, 4, 6 and 8 hours after drug administration on Days 1 and 8 of Cycle 1
Overall Response Rate (ORR)
Time Frame: Up to 60 months
Assessed using the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or Response Assessment in Neuro-Oncology (RANO) as appropriate
Up to 60 months
Duration of Response (DOR)
Time Frame: Up to 60 months
Up to 60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 4, 2014

Primary Completion (Actual)

February 1, 2017

Study Completion (Actual)

April 9, 2021

Study Registration Dates

First Submitted

April 16, 2014

First Submitted That Met QC Criteria

April 24, 2014

First Posted (Estimated)

April 25, 2014

Study Record Updates

Last Update Posted (Actual)

February 5, 2026

Last Update Submitted That Met QC Criteria

February 3, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20288
  • LOXO-TRK-14001 (Other Identifier: Loxo Inc.)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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