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)
12 Months
17 Years
Both
Anaplastic Lymphoma Kinase (ALK)
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 Location | Los Angeles, California 90095 |
| Children's Healthcare of Atlanta Dept of Oncology | Atlanta, Georgia 30342 |
| Dana Farber Cancer Institute Dept of Onc | Boston, Massachusetts 02115 |
| Memorial Sloan Kettering Cancer Center SC - 7 | New York, New York 10021 |
| Cincinnati Children's Hospital Medical Center Dept of Oncology | Cincinnati, Ohio 45229-3039 |
| St. Jude's Children's Research Hospital Dept of Oncology | Memphis, Tennessee 38105-2794 |
| Texas Children's Hospital Dept of Oncology | Houston, Texas 77030 |
