A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children (NCT02637687)

🧪 Interventions in This Study

• Larotrectinib (Vitrakvi, BAY2757556) (DRUG)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. Phase 1 (Closed):
2. 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.
3. 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.
4. 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.
5. Patients with primary CNS tumors or cerebral metastasis
6. Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
7. Adequate hematologic function
8. Adequate hepatic and renal function

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (45 Sites)

Showing the first 25 of 45 sites.

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

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

Explore by condition: All Solid Tumors Harboring NTRK Fusion clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Review the official NIH record below to find the coordinating site.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.