Patients & Caregivers

Our Clinical Trials

Our Clinical Trials

We are actively recruiting patients to a Phase 2 adult clinical trial, a Phase 1 adult clinical trial and a Phase 1/2 pediatric clinical trial for larotrectinib (LOXO-101).

We are grateful to the patients, families and physicians who have decided to contribute to our collective medical knowledge through the exploration of clinical trial options.

For more information about any of the larotrectinib (LOXO-101) clinical trials, please refer to or Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123.

Larotrectinib (LOXO-101), Loxo Oncology’s lead therapy, is an oral, selective inhibitor of tropomyosin receptor kinase (TRK), a family of signaling proteins that play an important role in cellular communication and tumor growth. TRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body.

Patients with advanced cancer who seek out tumor profiling or comprehensive cancer genetic testing may discover that their tumor harbors a TRK fusion, as this genetic event has been described across many tumor types, including:

  • acute myeloid leukemia
  • astrocytoma
  • brain low-grade glioma
  • breast cancer
  • colorectal cancer
  • congenital mesoblastic nephroma
  • gastrointestinal stromal tumors
  • glioblastoma multiforme
  • head and neck squamous cell cancer
  • intrahepatic cholangiocarcinoma
  • lung cancer
  • mammary analogue secretory carcinoma of salivary gland origin
  • melanoma
  • sarcoma
  • secretory breast cancer
  • thyroid cancer

Larotrectinib (LOXO-101) was purposely built to directly target TRK, and nothing else, turning off the signaling pathway that allows TRK fusion cancers to grow. Larotrectinib (LOXO-101) is the only selective TRK inhibitor in clinical development and is being studied in multiple clinical trials described below.

Larotrectinib (LOXO-101) is currently being evaluated in an ongoing multicenter, open-label, Phase 1 study in adult subjects with advanced solid tumors that have progressed or are non responsive to available therapies and for which no standard or available curative therapy exists.

In October 2015, we initiated a global, multi-center, single-arm, open-label Phase 2 basket trial in adult patients with solid tumors harboring TRK fusions. As a basket trial, the Phase 2 trial enrolls patients regardless of where their cancer arises in the human body, so long as the cancer harbors a TRK gene fusion.

In December 2015, we initiated a multicenter, open-label Phase 1 trial in pediatric patients with advanced solid or primary CNS tumors. The trial utilizes a liquid formulation of larotrectinib (LOXO-101) designed specifically for pediatric patients unable to swallow capsules.