A Phase I Study of LDK378 in Children with Cancers Containing an ALK Mutation

Protocol
13-235
Full 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)(Novartis CLDK378X2103)
Phase
I
Disease Status
Relapsed/Refractory
Purpose

Mutations of the ALK gene may fuel cancer growth. LDK378 is an investigational drug that may inhibit the growth of tumors containing an ALK mutation.

In this study, researchers want to find the highest dose of LDK378 that can be given safely in children with tumors that contain ALK mutations. Most patients in this study will have neuroblastoma, anaplastic large cell lymphoma, inflammatory myofibroblastoma, or rhabdomyosarcoma. LDK378 is a capsule that is taken orally (by mouth).

Eligibility

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • This study is for patients between the ages of 1 and 17 years.
  • Patients must have a locally advanced or metastatic cancer with an ALK mutation.
  • Patients must have a cancer that has continued to grow despite standard therapy or for which no standard therapy exists.
  • At least 3 weeks must pass between completion of previous chemotherapy and 2 weeks since prior radiation therapy or major surgery and entry into the study.

For more information about this study and to inquire about eligibility, please contact Dr. Shakeel Modak at 212-639-7623.

Disease(s)
Lymphoma: Non-Hodgkin's Lymphoma
Neuroblastoma
Sarcomas: Rhabdomyosarcoma
Solid Tumors
Locations