ADVL0319 PHASE I STUDY OF CC-5013 (LENALIDOMIDE) IN PEDIATRIC PATIENTS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This phase I study tests the safety of a new experimental drug called CC-5013, or lenalidomide in solid tumor patients and in patients with myelodysplastic syndrome (MDS). CC-5013 works to stop cancer cells from growing by blocking certain cell processes, causing the cell to die. Studies to find a safe dose of CC-5013 in adults are ongoing in Phase I trials like this one, but there have been no trials of CC-5013 in children yet. The goals of this study are to test the safety of CC-5013, to see what effects (good and bad) it has on children and children¿¿s cancer, and to find the highest dose of CC-5013 that can be given to children with solid tumors without causing severe side effects. Other goals of this study are to learn how the body handles the drug, and to better understand the functions in tumor cells that differ from healthy cells. The anti tumor effect of CC-5013 in solid tumor and MDS patients will also be studied. Because the main purpose of this phase I trial is to find the maximum dose of CC-5013 that can be safely given to children with solid tumors, the dose that some patients receive may be too small to have any effect on their cancer, or it may be too high and cause bad side effects. It is not known if this drug, at any dose, will be effective in treating cancer. Children with solid tumors will be separated into groups. The first group of children will be given CC-5013 at the starting dose. If the side effects at the starting dose are not too serious, the next group of children will be given a higher dose of CC-5013. This will continue until the maximum dose is found that does not result in unacceptable side effects. The dose of CC-5013 given will not be increased in each individual child. Children with MDS will not participate in the dose escalation portion of the protocol. Instead all patients with MDS will be treated with the same dose of CC-5013. The dose of CC-5013 that will be administered to patients with MDS is approximately equivalent to the dose that has been demonstrated to have clinical effect in adult patients with MDS. Each course of therapy will last for 28 days. During each course, the patient will be given CC-5013 by swallowing capsules, or by eating the contents of CC-5013 capsules sprinkled onto applesauce. CC-5013 will be given to children every day for 21 days, with a period of 7 days to rest before the next course begins. Patients on this study can be given CC-5013 as long as their cancer is not getting worse, and as long as no bad side effects happen, for up to 12 courses Children on this study will be given standard medical exams during therapy, such as physical exams, blood tests, and scans or other tests to evaluate their tumor. In addition to standard tests, patients will be the given the option to have blood samples taken for tests called pharmacokinetics to find out how the drug is behaving in cells the body, and for tests to learn what CC-5013 does to immune functions.