ALK-activated Tumors Clinical Trial
Official title:
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)
The purpose of this study was to estimate the maximum tolerated dose and/or recommended dose for expansion of LDK378 as a single agent, assess safety, tolerability and anti-tumor activity and characterize single and multiple-dose pharmacokinetics when administered orally to pediatric patients with ALK-activated tumors, with and without food.
LDK378 is a novel inhibitor of ALK that is active in a broad range of ALK-activated tumor
models, including models driven by mutated versions of ALK known to be resistant to
crizotinib, and by ALK gene amplification.
The primary purpose of this study was to determine the maximum tolerated dose and/or
recommended dose for expansion in pediatric patients, and to delineate a clinical dose to be
used in any future pediatric studies, with and without food. This study also assessed the
safety, tolerability, PK and preliminary evidence of antitumor activity of LDK378 in
pediatric patients with neuroblastoma, and other ALK-activated tumors.
Fasted cohort: each daily dose of LDK378 (including days which involved PK blood sampling)
was taken at least 2 hours after last meal & subjects did not eat until 1 hour after LDK378
was taken. Each daily dose of LDK378 was taken with 1-2 tablespoons (15-30 mL) of an
appropriate food (such as applesauce or non-fat yogurt) & a glass of water
Fed cohort: each daily dose of LDK378 (including days which involved PK blood sampling) was
taken with, or within 30 minutes after finishing a low-fat light snack containing 100-300
calories & 1.5-2 grams of fat.
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