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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)


Phase 1
12 Months
17 Years
Not Enrolling
Both
Anaplastic Lymphoma Kinase (ALK)

Thank you

Trial Information

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)


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 is 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. This study will also assess the safety, tolerability,
PK and preliminary evidence of antitumor activity of LDK378 in pediatric patients with
neuroblastoma, and other ALK-activated tumors.


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.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Incidence rate of Dose Limiting Toxicities (DLT)

Outcome Description:

cycle = within the first 21 days of patient's first dose

Outcome Time Frame:

up to day 21 after the patient's first dose

Safety Issue:

Yes

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CLDK378X2103

NCT ID:

NCT01742286

Start Date:

March 2013

Completion Date:

September 2015

Related Keywords:

  • Anaplastic Lymphoma Kinase (ALK)
  • pediatric, anaplastic lymphoma kinase, ALK-activated tumors, neuroblastoma, rhabdomyosarcoma, anaplastic large-cell lymphoma, inflammatory myofibroblastic tumor
  • Lymphoma

Name

Location

University of California at Los Angeles UCLA LeConte LocationLos Angeles, California  90095
Children's Healthcare of Atlanta Dept of OncologyAtlanta, Georgia  30342
Dana Farber Cancer Institute Dept of OncBoston, Massachusetts  02115
Memorial Sloan Kettering Cancer Center SC - 7New York, New York  10021
Cincinnati Children's Hospital Medical Center Dept of OncologyCincinnati, Ohio  45229-3039
St. Jude's Children's Research Hospital Dept of OncologyMemphis, Tennessee  38105-2794
Texas Children's Hospital Dept of OncologyHouston, Texas  77030