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

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 Protocol CLDK378X2103

Categories:

Solid Tumor

Phase I/II

Diseases Treated:

Refractory or progressive malignancies

Eligibility Overview:

  • Age > 12 months and < 18 years.
  • Participant has been diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or which no effective standard therapy exists.
  • Specimen is being submitted for molecular pre-screening to test for genetic alteration of ALK and the tumor must carry a genetic alteration of the ALK gene.
  • Participant has adequate liver, kidney, cardiac, and bone marrow function.

Description

This is a phase I study of a drug called LDK378. This is called a Phase I study because the goal is to find the highest dose of LDK378 that can be given safely. This drug will be given every day by mouth. This drug should be taken about the same time every day. There are two parts to this phase I study, a dose finding part and a dose expansion part.

In the dose finding part, a group of children will receive LDK378. If during the first 21 days of starting LDK378 no significant problems are seen, a second group of children will be given a higher dose of LDK378. This increase in doses will continue until the highest dose of LDK378 that is safe is found. In the dose expansion part, patients will receive the highest dose of LDK378 found to be safe in the dose finding part of the study.

Biomarker research will be a required part of this study. Biomarkers are important biological “indicators” that can be measured from tumor samples. In addition, tumor sample may be tested to try to learn more about the tumor, and to understand how the drug may be working in cancer.

Objectives

  • Find out what LDK378 does to the body, and find out what the body does to remove the drug (pharmacology research studies).
  • Learn more about other biomarkers (substances in the tumor that may help researchers understand the tumor better or give an indication of how the drug works in the cancer).

Eligibility criteria, among others, include:

  • Age > 12 months and < 18 years.
  • Participant has been diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or which no effective standard therapy exists.
  • Specimen is being submitted for molecular pre-screening to test for genetic alteration of ALK and the tumor must carry a genetic alteration of the ALK gene.
  • Participant has adequate liver, kidney, cardiac, and bone marrow function.

For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE (1-866-278-5833).

Contact

Alberto Pappo, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

Referring or consulting clinicians only: protocolinfo@stjude.org
For all other inquiries about St. Jude Children's Research Hospital studies: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.