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LDK378: A Phase I open-label, dose escalation study of LDK378

LDK378: A Phase I open-label, dose escalation study of LDK378 in pediatric patients with malignancies that have a genetic alteration in anaplastic lymphoma kinase (ALK)


Patients must be diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or for which no effective standard therapy exists. Tumor must carry a genetic alteration of ALK, such as a mutation, translocation, or amplification. Testing for ALK status can be done as part of the protocol screening process.


LDK378 is an orally available ALK inhibitor, approximately 20-fold more potent than crizotinib, and is more selective for ALK. It is anticipated the LDK378 may be more active than crizotinib in ALK-driven diseases, and may also be active in the setting of crizotinib-resistant disease. LDK378 will be administered once daily by mouth, NG tube, or G tube on an empty stomach.

Eligibility Criteria

  • Patients must be between 12 months and 17 years of age.
  • Patients with symptomatic CNS metastases must be neurologically stable and not requiring increasing doses of steroids or local CNS-directed therapy to control CNS disease.
  • Must not have clinically significant, uncontrolled heart disease or prolonged QTc interval.
  • Presence of any toxicities due to prior cancer therapy must be mild and tolerable.
  • Must have adequate organ function as evidenced by laboratory criteria.
  • Cannot have impairment of gastrointestinal function or GI disease that may significantly alter the absorption of LDK378 (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
  • No evidence of active viral hepatitis or a known diagnosis of HIV infection (testing not required).
  • Must not require therapy with any medications that are strong inhibitors or inducers of CYP3A4/5, substrates of CYP3A4/5 or CYP2C9 with narrow therapeutic indices, or medications known to prolong the QT interval or induce Torsades de Pointes.


For the current status and more detailed eligibility criteria of these clinical studies & protocols, referring physicians and families may call the Developmental Therapeutics Program directly at (832) 824-4266 at any time.