Oral TRK Inhibitor LOXO-101 for Treatment of Advanced Pediatric Solid or Primary Central Nervous System Tumors

Status: Recruiting
Phase: Phase 1
DFCI Protocol ID: 16-094

This is a multicenter, open-label, Phase 1 study in pediatric patients with advanced solid or primary CNS tumors. LOXO‑101 will be administered orally (PO) twice daily (BID), with the dose adjusted by body surface area (BSA).

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Steven Dubois MD, Dana Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria:

  -  Pediatric patients ≥ 1 year old on Cycle 1 Day 1 (C1D1)

  -  Locally advanced or metastatic solid or primary CNS tumor that has progressed, or was
     nonresponsive to available therapies, and for which no standard or available curative
     therapy exists

  -  Or patients ≥ 1 month old with a diagnosis of infantile/congenital fibrosarcoma, with
     a documented NTRK fusion that has progressed, or was nonresponsive to available
     therapies and for which no standard or available curative therapy exists

  -  Karnofsky (those 16 years old or older) or Lansky (those younger than 16 years)
     performance score of at least 50

  -  Adequate hematologic function: Absolute neutrophil count (ANC) ≥ 1.0 109/L, platelet
     count ≥ 100.0 109/L and hemoglobin ≥ 8.0 g/dL (patients with bone marrow involvement
     will not be evaluable for hematologic DLT and can enroll with ANC ≥ 0.75 109/L,
     platelet count ≥ 75.0 109/L and hemoglobin ≥ 8.0 g/dL)

  -  Adequate hepatic function: Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 upper
     limit of normal (ULN) for age (patients with documented Gilbert's Disease may be
     enrolled with Sponsor approval) and SGPT (ALT) ≤ 135 U/L. For the purpose of this
     study, the ULN for SGPT is 45 U/L

  -  Adequate renal function: Estimated glomerular filtration rate ≥ 30 mL/minute using
     the Cockroft-Gault formula or: a serum creatinine based on age/gender as outlined in
     the protocol

Exclusion Criteria:

  -  Investigational agent, anticancer therapy, or major surgery within 28 days (4 weeks)
     prior to C1D1

  -  Symptomatic brain metastases, leptomeningeal carcinomatosis, or spinal cord

  -  Clinically significant active cardiovascular disease or history of prolonged QT
     interval corrected for heart rate (QTc)

  -  Current treatment with a strong cytochrome P450 (CYP) 3A4 inhibitor or inducer
     (EIAEDs and dexamethasone for CNS tumors or metastases, on a stable dose, are

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