The purpose of this research study is to evaluate a new investigational drug (TPI 287) for neuroblastoma and medulloblastoma. An investigational drug is one that has not yet been approved by the Food and Drug Administration. This investigational drug is called TPI 287. This study will look at the tumor's response to the study drug, TPI 287, as well as the safety and tolerability of the drug. TPI 287 was shown to be effective in stopping tumor growth and was also shown to be safe in three different animal species. TPI 287 has been tested in humans in four clinical trials, and approximately 100 subjects with various types of cancers have received the drug, including a pediatric population in our previous Phase I trial.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Rady Children's Hospital
San Diego, California, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Phase I portion of trial- To determine the safety and tolerability of TPI 287 as a single agent in pediatric and young adult patients with refractory or recurrent neuroblastoma or medulloblastoma. Adverse events collected from time of first dose to 30 days past last dose and until all related events resolved, average of one year.
Time frame: length of study +30 days
Number of Participants With Overall Response Assessed Using RECIST Criteria
Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Time frame: 6 months
Number of Days Participants Experienced Progression Free Survival (PFS)
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Time frame: 3 years
Median Overall Survival (OS) of Participants
Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
Time frame: 3 years
Quality of Life of Children Receiving TPI287 Using PedsQL Questionnaires
To evaluate the impact of QOL of children receiving TPI287 using PedsQL questionnaires
Time frame: 3 years
To Evaluate the Drug Levels and Pharmacokinetics (PK) of TPI 287 From Blood Samples at Multiple Time Points Within the First 24 Hours on Study.
To evaluate the pharmacokinetics (PK) of TPI 287 in the Phase I population of this trial.
Time frame: 1 year
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