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

Giselle Sholler

Status and phase

Terminated

Phase 2

Phase 1

Conditions

Neuroblastoma

Medulloblastoma

Treatments

Drug: TPI 287

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01483820

NMTRC 004

Details and patient eligibility

About

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.

Enrollment

8 patients

Sex

All

Ages

12 months to 30 years old

Volunteers

No Healthy Volunteers

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