Trial record 1 of 1 for:    NMTRC004
Previous Study | Return to List | Next Study

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Spectrum Health Hospitals
Sponsor:
Collaborators:
Van Andel Research Institute
Cortice Biosciences, Inc.
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT01483820
First received: November 28, 2011
Last updated: February 5, 2014
Last verified: February 2014
  Purpose

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 Intervention Phase
Neuroblastoma
Medulloblastoma
Drug: TPI 287
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Trial of TPI-287 in Patients With Refractory or Recurrent Neuroblastoma and Medulloblastoma

Resource links provided by NLM:


Further study details as provided by Spectrum Health Hospitals:

Primary Outcome Measures:
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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)


Secondary Outcome Measures:
  • Determine the Overall Response Rate (ORR) of Participants using RECIST criteria [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    To evaluate the pharmacokinetics (PK) of TPI 287 in the Phase I population of this trial.


Estimated Enrollment: 114
Study Start Date: November 2011
Estimated Study Completion Date: November 2018
Estimated Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   12 Months to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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- http://ctep.cancer.gov/forms/CTCAEv4.pdf)
  • 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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01483820

Contacts
Contact: Genevieve Bergendahl, RN 616-267-0335 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa v (616) 267-0327 alyssa.vanderwerff@helendevoschildrens.org

Locations
United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: Mehrzad Milburn    858-966-8155      
Principal Investigator: William Roberts, MD         
United States, Connecticut
Connecticut Children's Hospital Recruiting
Hartford, Connecticut, United States, 06106
Contact: Jennifer Hylton    860-545-9337    Jhylton02@connecticutchildrens.org   
Principal Investigator: Nehal Parikh, MD         
United States, Florida
Arnold Palmer Hospital for Children- MD Anderson Recruiting
Orlando, Florida, United States, 32806
Contact: Michelle Pope, RN    321-841-8588      
Principal Investigator: Don Eslin, MD         
United States, Michigan
Helen DeVos Children's Hospital Recruiting
Grand Rapids, Michigan, United States, 49503
Contact: Shannon Mackeigan, N    616-267-1162    shannon.mackeigan@helendevoschildrens.org   
Principal Investigator: Deanna Mitchell, MD         
Principal Investigator: Giselle Sholler, MD         
United States, Missouri
Children's Mercy Hospitals and Clinics Recruiting
Kansas City, Missouri, United States, 64108
Contact: Sara Soliman, RN    816-855-1977    sjsoliman@cmh.edu   
Principal Investigator: Kathleen Neville, MD         
Cardinal Glennon Children's Medical Center Not yet recruiting
St. Louis, Missouri, United States, 63104
Contact: Katherine Maxwell, RN    314-268-4000      
Principal Investigator: William Ferguson, MD         
United States, North Carolina
Levine Children's Hospital Recruiting
Charlotte, North Carolina, United States, 28204
Contact: Nicole Turner, RN    980-442-2355    felisha.turner@carolinashealthcare.org   
Principal Investigator: Javier Oesterheld, MD         
United States, South Carolina
Medical University of South Carolina Not yet recruiting
Charleston, South Carolina, United States, 29425
Contact: Jacqueline Kraveka, MD    843-792-2957      
Principal Investigator: Jaqueline Kraveka, MD         
Sponsors and Collaborators
Giselle Sholler
Van Andel Research Institute
Cortice Biosciences, Inc.
Investigators
Study Chair: Nehal Parikh, MD Connecticut Children's Hospital
Principal Investigator: Giselle Sholler, MD The Spectrum Health Group
  More Information

Additional Information:
No publications provided

Responsible Party: Giselle Sholler, Vice Study Chair, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01483820     History of Changes
Other Study ID Numbers: NMTRC 004
Study First Received: November 28, 2011
Last Updated: February 5, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Medulloblastoma
Neuroblastoma
Glioma
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neuroectodermal Tumors, Primitive
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neuroectodermal Tumors, Primitive, Peripheral

ClinicalTrials.gov processed this record on July 29, 2014