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 also actively recruiting patients to a Phase 1 clinical trial for LOXO-292.

Larotrectinib (LOXO-101)

Larotrectinib (LOXO-101), Loxo Oncology’s lead investigational 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.

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.

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

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

LOXO-292 (RET inhibitor)

LOXO-292 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. 

RET fusions have been identified in approximately 2% of non-small cell lung cancer, approximately 10-20% of papillary thyroid cancer, and a subset of colon and other cancers. RET point mutations account for approximately 60% of medullary thyroid cancer. Both RET fusion and select RET mutated cancers are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as “oncogene addiction,” renders such tumors highly susceptible to small molecule inhibitors targeting RET.

LOXO-292 was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms that could otherwise limit the activity of this therapeutic approach.

For more information about the LOXO-292 clinical trial, please refer to clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology RET Physician and Patient Clinical Trial Hotline for more information about the LOXO-292 clinical trial at 1-855-RET-4-292 or email clinicaltrials@loxooncology.com.


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.


LOXO-292, a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase, is currently being evaluated in a global, multi-center Phase 1 trial in patients with advanced solid tumors.