To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With Refractory Cancer

Completed · Phase 1

Conditions studied: Sarcoma, Neuroblastoma, Wilms Tumor, Leukemia, Lymphoma, Brain Tumors

In brief

Background: * Marqibo(Registered Trademark) is a new anticancer drug. It combines Vincristine sulfate, which is a widely used anticancer drug, and packages it into a tiny fat bubble known as a liposome. The goal of this is to improve the drug's ability to destroy cancer cells and help reduce the potential side effects of treatment. * Vincristine sulfate was originally developed from chemicals found in the periwinkle plant and acts against multiple types of malignant cancer. It is approved for multiple cancer types including solid tumors and blood cancers. * Research has shown that Marqibo(Registered Trademark) is able to slow or stop the growth of cancer cells in some adults, both alone and in combination with other chemotherapy drugs, but more research is needed to determine its use in children. * There has been one previous small study of Marqibo(Registered Trademark) in children. Although some anti-cancer activity was seen, side effects and optimal dosing were not fully determined. * As is seen with standard Vincristine suflate, the most common side effect of Marqibo(Registered Trademark) involves the nervous system. It can cause numbness and tingling in the hands and feet. Symptoms commonly improve when the drug is discontinued or the dose is lowered. Objectives: \- To determine the safety and efficacy of Marqibo as a treatment for children who have been diagnosed with certain types of malignant cancer that has not responded to standard treatment. Eligibility: * Children and adolescents between 2 and 21 years of age who have been diagnosed with certain types of malignant cancer that has not responded to standard treatment. * These cancer types include solids tumors, primary brain tumors, leukemias, and lymphomas.

Key facts

Study ID

NCT01222780

Run by

Acrotech Biopharma Inc.

People needed

22

Starts

2010-09-01

Expected to finish

2014-12-01

Last updated by the study team

2020-01-18

Who can join

Age: 2 and older, up to 20. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Clinically significant unrelated systemic illness, such as uncontrolled serious infection or organ dysfunction, which in the judgment of protocol investigators would compromise the subject s ability to tolerate the investigational agent or interfere with the study procedures or results. This includes any condition or circumstance that in the opinion of the investigator would significantly interfere with the subject s protocol compliance and put the subject at increased risk.
• CNS leukemia or lymphoma as manifested by any of the following:
• CSF WBC > 5/microL and confirmation of CSF blasts.
• Cranial neuropathies deemed secondary to underlying malignancy
• CT or MRI scan evidence.
• Note: History of CNS involvement is not an exclusion criterion.
• Neutrophil count < 1,000/microL or platelet count of < 50,000/microL (except for the expanded ALL cohort, where there is no blood count requirement).
• Pregnant or breast-feeding females are excluded because Marqibo(Registered Trademark) may be harmful to the developing fetus or nursing child.
• Currently receiving other investigational agents.
• History of previously receiving Marqibo(Registered Trademark).
• History of allergic reactions or serious adverse events attributed to compounds of similar chemical or biologic composition to vincristine or components of Marqibo(Registered Trademark) (vincristine sulfate injection, sphingomyelin/cholesterol liposomes, sodium phosphate injection).
• Are eligible for stem cell transplantation. This implies that a suitable donor is readily available, that the primary oncology team and Principal Investigator recommend this as a preferred treatment option at the time of protocol evaluation, and that the subject is willing to undergo stem cell transplantation.
• Presence of preexisting Grade 2 or greater sensory or motor neuropathy.
• History of persistent greater than or equal to Grade 2 active neurologic disorders unrelated to chemotherapy.
• Positive for human immunodeficiency virus (HIV) due to the increased risk of complications.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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