Clinical Trial: Safety and Efficacy Study of TPI-287 in Neuroblastoma and Medulloblastoma

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: A Phase I/II Trial of TPI-287 in Patients With Refractory or Recurrent Neuroblastoma and Medulloblastoma

Brief Summary:

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.


Detailed Summary:
Sponsor: Giselle Sholler

Current Primary Outcome: Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: length of study +30 days ]

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.


Original Primary Outcome:

  • Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 months ]
    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
  • Determine the Overall Response Rate (ORR) of Participants using RECIST criteria [ Time Frame: 3 years ]
    Phase II portion of trial- To determine the activity of TPI 287 in these tumor types based on Overall Response Rate (ORR), including complete and partial responses (CR and PR)


Current Secondary Outcome:

  • Number of Participants With Overall Response Assessed Using RECIST Criteria [ Time Frame: 6 months ]
    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.
  • Number of Days Participants Experienced Progression Free Survival (PFS) [ Time Frame: 3 years ]
    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.
  • Median Overall Survival (OS) of Participants [ Time Frame: 3 years ]
    Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
  • Quality of Life of Children Receiving TPI287 Using PedsQL Questionnaires [ Time Frame: 3 years ]
    To evaluate the impact of QOL of children receiving TPI287 using PedsQL questionnaires
  • 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. [ Time Frame: 1 year ]
    To evaluate the pharmacokinetics (PK) of TPI 287 in the Phase I population of this trial.


Original Secondary Outcome:

  • Determine the Overall Response Rate (ORR) of Participants using RECIST criteria [ Time Frame: 6 months ]
    Phase I portion of trial- To determine the activity of TPI 287 in these tumor types based on Overall Response Rate (ORR), including complete and partial responses (CR and PR)
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 3 years ]
    Phase II 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
  • Determine the Progression Free Survival (PFS) of Participants using days until progression [ Time Frame: 3 years ]
    To determine the activity of TPI 287 based on Progression Free Survival (PFS) in pediatric and adolescent subjects with refractory or recurrent neuroblastoma and medulloblastoma
  • Determine the Median overall survival (OS) of Participants [ Time Frame: 3 years ]
    Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
  • Evaluate the impact of Quality of Life of children receiving TPI287 using PedsQL questionnaires [ Time Frame: 3 years ]
    To evaluate the impact of QOL of children receiving TPI287
  • Evaluate a descriptive assessment of pain scores in patients receiving TPI287 using Pain diaries and morphine equivalence scores [ Time Frame: 3 years ]
    To evaluate a descriptive assessment of pain scores in patient receiving TPI287
  • 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. [ Time Frame: 1 year ]
    To evaluate the pharmacokinetics (PK) of TPI 287 in the Phase I population of this trial.


Information By: Spectrum Health Hospitals

Dates:
Date Received: November 28, 2011
Date Started: December 2011
Date Completion:
Last Updated: September 9, 2016
Last Verified: September 2016