Larotrectinib (LOXO-101) Trials
Call us to find out more information about NTRK fusion testing, larotrectinib (LOXO-101), and clinical trial access.
Clinical Trial Hotline
Loxo Oncology’s lead investigational agent for TRK inhibition,
Loxo Oncology’s lead therapy in clinical development,
Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally bound 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
- Brain low-grade glioma
- Breast cancer
- Colorectal cancer
- Congenital mesoblastic nephroma
- Gastrointestinal stromal tumors
- Glioblastoma multiforme
- Head and neck squamous cell cancer
- Infantile fibrosarcoma
- Intrahepatic cholangiocarcinoma
- Lung cancer
- Mammary analog secretory carcinoma of salivary gland origin
- Secretory breast cancer
- Thyroid cancer
Larotrectinib (LOXO-101) was designed to target TRK, turning off the signaling pathway that allows TRK fusion cancers to grow. Larotrectinib (LOXO-101) was developed in partnership with Array BioPharma.
Across three pivotal clinical trials, larotrectinib has demonstrated interim data showing 76% confirmed objective response rate in 55 patients across 17 tumor types in TRK fusion adult and pediatric cancers.
- Interim analysis included 55 patients across 17 tumor types pooled from 3 ongoing trials; 50 of these patients were evaluable for confirmed response
- 93% of responding patients remain on larotrectinib or received surgery with curative intent
- The safety data presented encompass the entire larotrectinib safety database in cancer patients (n = 125). The most common treatment-emergent adverse events, regardless of relationship to larotrectinib, included fatigue (15% grade 1, 18% grade 2, 5% grade 3), dizziness (22% grade 1, 4% grade 2, 2% grade 3), nausea (20% grade 1, 5% grade 2, 2% grade 3), and anemia (8% grade 1, 9% grade 2, 9% grade 3). Seven (13%) of the 55 patients with TRK fusion cancers required a dose reduction due to an adverse event. None of the 55 patients with TRK fusion cancers discontinued larotrectinib due to an adverse event
- Median duration of response and progression-free survival end points have not yet been met
Both the FDA and EMA have granted orphan drug designation for
Global, multicenter, single-arm, open-label, Phase 2 basket trial in patients with solid tumors with a fusion of NTRK1, NTRK2, or NTRK3.
Loxo Oncology collaborates with the clinical, laboratory, and molecular pathology communities in both academia and industry to help ensure that patients with TRK fusions and their treating physicians are notified about the larotrectinib (
This Phase 1/2 clinical trial is a multicenter, open-label trial in pediatric patients with advanced solid or primary central nervous system tumors. The trial uses a liquid formulation of larotrectinib (
1. Hyman DM, et al. The efficacy of larotrectinib (LOXO-101), a selective tropomyosin receptor kinase (TKR) inhibitor, in adult and pediatric TRK fusion cancers. In: Proceedings from the 2017 ASCO Annual Meeting; June 2–6, 2017; Chicago, Illinois. Abstract LBA2501. 2. Laetsch TW, et al. A pediatric phase I study of larotrectinib, a highly selective inhibitor of the tropomyosin receptor kinase (TRK) family. In: Proceedings from the 2017 ASCO Annual Meeting; June 2–6, 2017; Chicago, Illinois. Abstract 10510.
For additional information about any of the
Loxo Oncology works with a patient concierge service to help ensure that travel and logistics for patients and caregivers are not a barrier to trial entry.
Interested physicians and patients can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 (1-855-687-5123).
Loxo Oncology is committed to helping patients who have not responded to available therapies and may benefit from its investigational therapies. Loxo Oncology's Policy for Access to Investigational Agents describes the principles and government regulations that the company will follow when considering a request.