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Safety and Efficacy Study of TPI-287 in Neuroblastoma and Medulloblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01483820
Recruitment Status : Terminated (Lack of enrollment)
First Posted : December 1, 2011
Results First Posted : October 28, 2016
Last Update Posted : April 22, 2022
Cortice Biosciences, Inc.
Information provided by (Responsible Party):
Wake Forest University Health Sciences

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.

Condition or disease Intervention/treatment Phase
Neuroblastoma Medulloblastoma Drug: TPI 287 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Trial of TPI-287 in Patients With Refractory or Recurrent Neuroblastoma and Medulloblastoma
Study Start Date : December 2011
Actual Primary Completion Date : December 2014
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Arm Intervention/treatment
Experimental: TPI 287
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.
Drug: TPI 287
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.

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. Median Overall Survival (OS) of Participants [ Time Frame: 3 years ]
    Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)

  4. 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

  5. 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 from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Months to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subjects must have histologically proven neuroblastoma or medulloblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
  • Subjects must be age >12 months and diagnosed before the age of 21
  • Measurable disease, including at least one of the following:
  • Measurable tumor >10mm by CT or MRI
  • Positive bone marrow biopsy/aspirate.
  • Positive MIBG
  • Current disease state must be one for which there is currently no known curative therapy
  • Lansky Play Score or Karnofsky scale must be more than 30
  • Subjects without bone marrow metastases must have an ANC > 750/μl and platelet count >50,000/μl
  • Adequate Renal Function Defined As
  • Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 or
  • A serum creatinine based on age/gender
  • Adequate liver function must be demonstrated, defined as:
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
  • SGPT (ALT) < 10 x upper limit of normal (ULN) for age
  • SGOT (AST) < 10x upper limit of normal (ULN) for age
  • No other significant organ toxicity defined as > Grade 2 by National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI-CTCAE V4.0-
  • A negative urine pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
  • Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
  • Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines
  • Subjects may have received microtubulin inhibitors during previous therapies.

Exclusion Criteria:

  • Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the effects of prior chemotherapy (hematological and bone marrow suppression effects), generally at least 3 weeks from the most recent administration (6 weeks for nitrosoureas).
  • Subjects who have received any myeloablative therapy within the previous 2 months.
  • Subjects receiving anti-tumor therapy for their disease or any investigational drug concurrently
  • Subjects with serious infection or a life-threatening illness (unrelated to tumor) that is > Grade 2 (NCI CTCAE V4.0), or active, serious infections requiring parenteral antibiotic therapy.
  • Subjects with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study
  • Subjects with known hypersensitivity to any of the components of the drugs to be administered on study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01483820

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United States, California
Rady Children's Hospital
San Diego, California, United States, 92123
United States, Connecticut
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
United States, Florida
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, United States, 32806
United States, Michigan
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
United States, Missouri
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Medical Center
Saint Louis, Missouri, United States, 63104
United States, North Carolina
Levine Children's Hospital
Charlotte, North Carolina, United States, 28204
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Wake Forest University Health Sciences
Cortice Biosciences, Inc.
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Study Chair: Nehal Parikh, MD Connecticut Children's Hospital
Principal Investigator: Giselle Sholler, MD Beat Childhood Cancer at Atrium Health
Additional Information:
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Responsible Party: Wake Forest University Health Sciences Identifier: NCT01483820    
Other Study ID Numbers: NMTRC 004
First Posted: December 1, 2011    Key Record Dates
Results First Posted: October 28, 2016
Last Update Posted: April 22, 2022
Last Verified: November 2020
Additional relevant MeSH terms:
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Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue