INCB18424 in Treating Young Patients With Relapsed or Refractory Solid Tumor, Leukemia, or Myeloproliferative Disease
- Conditions
- Myelodysplastic/Myeloproliferative NeoplasmsUnspecified Childhood Solid Tumor, Protocol SpecificChronic Myeloproliferative DisordersLeukemiaMyelodysplastic Syndromes
- Interventions
- Registration Number
- NCT01164163
- Lead Sponsor
- Children's Oncology Group
- Brief Summary
RATIONALE: INCB18424 (Ruxolitinib) may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
PURPOSE: This phase 1 clinical trial is studying the side effects and best dose of INCB18424 in treating young patients with relapsed or refractory solid tumor, leukemia, or myeloproliferative disease.
- Detailed Description
OBJECTIVES:
Primary
* To estimate the maximum-tolerated dose and/or recommended phase II dose of oral JAK inhibitor INCB18424 administered continuously, twice daily to pediatric patients with relapsed or refractory solid tumors.
* To define and describe the toxicities of this treatment administered on this schedule in pediatric patients with relapsed or refractory solid tumors, leukemias, or myeloproliferative neoplasms (MPNs).
* To characterize the pharmacokinetics of this treatment in pediatric patients with relapsed or refractory solid tumors, leukemias, or MPNs.
Secondary
* To preliminarily define the antitumor activity of this treatment within the confines of a phase I study.
* To assess the biologic activity of oral JAK inhibitor INCB18424 upon JAK-STAT signaling in pediatric patients with relapsed or refractory solid tumors, leukemias, or MPNs.
* To assess the cytotoxicity and biologic activity of oral JAK inhibitor INCB18424 upon phosphosignaling and mutation burden in pediatric patients whose leukemias or MPNs have known CRLF2 and/or JAK mutations.
OUTLINE: This is a multicenter, dose-escalation study.
Patients receive oral JAK inhibitor INCB18424 twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Patients with relapsed or refractory leukemia may receive intrathecal chemotherapy in course 2 and subsequent courses at the discretion of the treating physician.
Plasma, bone marrow, and blood samples may be collected at baseline, during course 1, and before subsequent courses for pharmacokinetic analysis and correlative biology studies.
After completion of study treatment, patients are followed up for 30 days.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 49
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Treatment (Ruxolitinib) ruxolitinib phosphate - Treatment (Ruxolitinib) pharmacological study - Treatment (Ruxolitinib) laboratory biomarker analysis -
- Primary Outcome Measures
Name Time Method Pharmacokinetics Up to 28 days Toxicity 30 days post treatment Maximum-tolerated dose and/or recommended phase II dose 28 days
- Secondary Outcome Measures
Name Time Method Toxicity and biologic activity Day 1 and Day 15 Antitumor activity Up to 30 days post treatment
Trial Locations
- Locations (24)
UAB Comprehensive Cancer Center
🇺🇸Birmingham, Alabama, United States
UCSF Helen Diller Family Comprehensive Cancer Center
🇺🇸San Francisco, California, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Hospital Colorado Center for Cancer and Blood Disorders
🇺🇸Aurora, Colorado, United States
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
🇺🇸Atlanta, Georgia, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
🇺🇸Baltimore, Maryland, United States
Children's Memorial Hospital - Chicago
🇺🇸Chicago, Illinois, United States
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office
🇺🇸Bethesda, Maryland, United States
Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Masonic Cancer Center at University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis
🇺🇸St. Louis, Missouri, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
C.S. Mott Children's Hospital at University of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
🇺🇸New York, New York, United States
Knight Cancer Institute at Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas
🇺🇸Dallas, Texas, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Baylor University Medical Center - Houston
🇺🇸Houston, Texas, United States
Children's Hospital and Regional Medical Center - Seattle
🇺🇸Seattle, Washington, United States
Midwest Children's Cancer Center at Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Riley's Children Cancer Center at Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States