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

NCT01483820 | Terminated Phase 1 Results DMC

• 1 other identifier: NMTRC 004
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 8

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.

Conditions

Neuroblastoma; Medulloblastoma

Outcome Measures

Primary Outcomes (1)

• 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.

  length of study +30 days

Secondary Outcomes (5)

• Number of Participants With Overall Response Assessed Using RECIST Criteria

  6 months

• Number of Days Participants Experienced Progression Free Survival (PFS)

  3 years

• Median Overall Survival (OS) of Participants

  3 years

• Quality of Life of Children Receiving TPI287 Using PedsQL Questionnaires

  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.

  1 year

Study Arms (1)

TPI 287

EXPERIMENTAL

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

Interventions

TPI 287 DRUG

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.

TPI 287

Eligibility Criteria

• Age: 12 Months - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Giselle Sholler: lead
• Cortice Biosciences, Inc.: collaborator

Study Sites (8)

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

Location

Arnold Palmer Hospital for Children- MD Anderson

Orlando, Florida, 32806, United States

Location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

Location

Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States

Location

Levine Children's Hospital

Charlotte, North Carolina, 28204, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Related Links

• Beat Childhood Cancer Consortium

MeSH Terms

Conditions

Neuroblastoma; Medulloblastoma

Interventions

TPI-287

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Glioma

Results Point of Contact

• Title: Giselle Sholler, MD
• Organization: NMTRC

giselle.sholler@helendevoschildrens.org

6162670335

Study Officials

• Nehal Parikh, MD

  Connecticut Children's Hospital

  STUDY CHAIR

• Giselle Sholler, MD

  Beat Childhood Cancer at Atrium Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Vice Study Chair

Study Record Dates

• First Submitted: November 28, 2011
• First Posted: December 1, 2011
• Study Start: December 1, 2011
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2014
• Last Updated: August 6, 2024
• Results First Posted: October 28, 2016

Record last verified: 2024-08

Locations

US (8)