Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)

Status: Recruiting
Phase: Phase 1
Diagnosis: Pediatric Lymphoma
NCT ID: NCT01742286 (View complete trial on
DFCI Protocol ID: 13-180


The purpose of this study is to estimate the maximum tolerated dose 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.


Conducting Institutions:
Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:
Suzanne Shusterman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

Inclusion Criteria: - Diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or for which no effective standard therapy exists - Age ≥ 12 months and ≤ 17 years - The tumor must carry a genetic alteration of ALK - Patients must have evaluable or measurable disease Exclusion criteria: - Symptomatic central nervous system (CNS) metastases who are neurologically unstable or require increasing doses of steroids or local CNS-directed therapy (such as radiotherapy, surgery or intrathecal chemotherapy) to control their CNS disease - Clinically significant, uncontrolled heart disease - Inadequate end organ function as defined by specified laboratory values - Use of medications that are known to be strong inhibitors or inducers of CYP3A4/5 that cannot be discontinued at least 1 week prior to start of treatment with LDK378 and for the duration of the study - Use of medications that are mainly metabolized by CYP3A4/5 or CYP2C9 that cannot be discontinued at least 1 week prior to start of treatment with LDK378 and for the duration of the study.
  • Email
  • Print
  • Share
  • Text
Highlight Glossary Terms
  • Make an Appointment

    • For adults:
      877-442-3324 (877-442-DFCI)

    • Quick access:
      Appointments as soon as the next day for new adult patients

    • For children:
      888-733-4662 (888-PEDI-ONC)

    • Make Appointment Online