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

Status: Recruiting
Phase: Phase 1
DFCI Protocol ID: 13-180

The purpose of this study is 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.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Suzanne Shusterman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Contacts:

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

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 < 18 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.

  -  History of interstitial lung disease or interstitial pneumonitis, including
     clinically significant radiation pneumonitis

  -  History of pancreatitis or history of increased amylase or lipase that was due to
     pancreatic disease.

  -  Medications with a known risk of prolongation of QT interval

Other protocol defined inclusion and exclusion criteria may apply

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