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Pediatric Cancers

Phase I Trial to Evaluate the Safety, Activity and Pharmacokinetics of Marqibo® (Vincristine Sulfate Liposomes Injection) in Children and Adolescents With Refractory Cancer

NCI-10-C-0220, NCT01222780

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Investigator(s):

Alan S. Wayne, M.D.
Principal Investigator
Phone: 301-496-4256
Fax: 301-451-7010
waynea@mail.nih.gov

Referral Contact(s):

Pediatric Oncology
Phone: 301-496-4256
1-877-624-4878 (Toll free)

 

Background:

  • Vincristine sulfate is a widely used antineoplastic agent of the alkaloid class of drugs derived from the periwinkle plant (Vinca rosea Linn.) having activity against a wide number of malignancies
  • Vincristine sulfate is a cell-cycle-specific agent that arrests cell growth in the M-phase (metaphase) by binding specifically with tubulin and disrupting cell division; its dose limiting toxicity (DLT) is virtually always neurological
  • Investigations over the past 2 decades have demonstrated that liposomal drug carriers are capable of increasing the therapeutic index of anticancer drugs by altering the drug's pharmacological behavior
  • Marqibo® is vincristine encapsulated in sphingomyelin/cholesterol (SM/CHOL) liposomes, developed to increase the activity of vincristine
  • Marqibo® has been shown to have activity in Phase 2 studies in adults
  • Previous investigation of Marqibo® in children is limited:
    • A small Phase 2 study was conducted with dosing of 2.0 mg/m2 every 14 days
    • Marqibo® appeared to be well tolerated and some activity was seen, although data were quite limited

Objectives:

  • Define the maximum tolerated dose (MTD), toxicity profile, dose-limiting toxicities, and pharmacokinetics in children and adolescents with solid tumors or hematologic malignancies receiving weekly intravenous doses of Marqibo®
  • Define the tolerability and potential activity of Marqibo® in children and adolescents with relapsed or refractory acute lymphoblastic leukemia (ALL) at the pediatric MTD

Key Eligibility Criteria:

  • Children and adolescents ≥ 2 years and < 21 years of age
  • Histologically confirmed relapsed or refractory malignant disease that is measurable or evaluable

Study Outline:

  • Marqibo® will be administered intravenously over 60 minutes (±10 minutes) every 7 days (±3 days) (Days 1, 8, 15, 22) for four doses (1 cycle)
  • Cycles may be repeated every 28 days for a maximum of 6 cycles; additional cycles may be offered with evidence of acceptable toxicity and clinical benefit
  • The trial follows a standard Phase I design, with three to six subjects per dose level and standard definitions of maximum tolerated dose (MTD) and dose limiting toxicity (DLT); at the MTD, a total of six additional subjects with relapsed or refractory acute lymphoblastic leukemia (ALL) will be evaluated
  • Detailed pharmacokinetic studies will be performed during the first treatment cycle

Additional Information:

  • This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
  • There is no charge for medical care received at NIH Clinical Center.
  • PDQ (Physicians Data Query) - provides additional details about this study for health care providers.
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Reviewed: 12/14/12
Updated: 12/8/10

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