A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Solid Tumors Harboring NTRK Fusion

In brief

The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer. The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug. The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with larotrectininb.

Key facts

Study ID

NCT02637687

Run by

Bayer

People needed

154

Starts

2015-12-16

Expected to finish

2026-09-30

Last updated by the study team

2025-06-22

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Phase 1 (Closed):
• Dose escalation: Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists; OR Infants from birth and older with a diagnosis of malignancy and with a documented NTRK fusion that has progressed or was nonresponsive to available therapies, and for which no standard or available curative therapy exists; OR Patients with locally advanced infantile fibrosarcoma who would require, in the opinion of the investigator, disfiguring surgery or limb amputation to achieve a complete surgical resection. Phase I dose escalation cohorts are closed to enrollment.
• Dose expansion: In addition to the above stated inclusion criteria, patients must have a malignancy with a documented NTRK gene fusion with the exception of patients with infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer. Patients with infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer may enroll into this cohort with documentation of an ETV6 rearrangement by FISH or RT-PCR or a documented NTRK fusion by next generation sequencing.
• Phase 2:
• - Infants from birth and older at C1D1 with a locally advanced or metastatic infantile fibrosarcoma, patients with locally advanced infantile fibrosarcoma who would require, in the opinion of the investigator, disfiguring surgery or limb amputation to achieve a complete surgical resection; OR Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists with a documented NTRK gene fusion (or in the case of infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer with documented ETV6 rearrangement (or NTRK3 rearrangement after discussion with the sponsor) by FISH or RT-PCR. Patients with NTRK-fusion positive benign tumors are also eligible; OR Potential patients older than 21 years of age with a tumor diagnosis with histology typical of a pediatric patient and an NTRK fusion may be considered for enrollment following discussion between the local site Investigator and the Sponsor.
• Patients with primary CNS tumors or cerebral metastasis
• Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
• Adequate hematologic function
• Adequate hepatic and renal function

You may not qualify if…

• Major surgery within 14 days (2 weeks) prior to C1D1
• Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to C1D1, ongoing cardiomyopathy; current prolonged QTc interval > 480 milliseconds
• Active uncontrolled systemic bacterial, viral, or fungal infection
• Current treatment with a strong CYP3A4 inhibitor or inducer. Enzyme-inducing anti-epileptic drugs (EIAEDs) and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed.
• Phase 2 only:
• Prior progression while receiving approved or investigational tyrosine kinase inhibitors targeting TRK, including entrectinib, crizotinib and lestaurtinib. Patients who received a TRK inhibitor for less than 28 days of treatment and discontinued because of intolerance remain eligible.

Where it is running

• Children's Hospital Los Angeles - Hematology/Oncology — Los Angeles, California, United States
• UCLA Jonsson Comprehensive Cancer Center — Los Angeles, California, United States
• Lucille Packard Children's Hospital Stanford - Pediatric Nephrology — Palo Alto, California, United States
• Nemours Children's Hospital - Florida - Hematology / Oncology — Orlando, Florida, United States
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States
• Memorial Sloan Kettering Cancer Center New York - Main Campus — New York, New York, United States
• Cincinnati Children's Hospital Medical Center | Division of Nephrology and Hypertension — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia - Hematology/Oncology — Philadelphia, Pennsylvania, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• University of Texas Southwestern Medical Center — Dallas, Texas, United States
• Seattle Children's Hosptial - Oncology — Seattle, Washington, United States
• Sydney Children's Hospital — Sydney, New South Wales, Australia
• Women's and Children's Hospital — North Adelaide, South Australia, Australia
• Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
• BC Children's Hospital - Hematology/Oncology — Vancouver, British Columbia, Canada
• The Hospital for Sick Children (SickKids) — Toronto, Ontario, Canada
• CHU Sainte-Justine — Montreal, Quebec, Canada
• Beijing Children's Hospital, Capital Medical University — Beijing, Beijing Municipality, China
• Sun Yat-sen University Cancer Center — Guangzhou, Guangdong, China
• Tianjin Medical University Cancer Institute & Hospital — Tianjin, China
• FN Brno - Detska nemocnice — Brno, Czechia
• Fakultni nemocnice v Motole — Prague, Czechia
• Rigshospitalet - Børn og Unge — Copenhagen, Denmark
• Institut Curie - Ulm - Paris — Paris, France
• Gustave Roussy - Departement Oncologie-Radiotherapie — Villejuif, France

Full record on ClinicalTrials.gov

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