Symptoms and General Pathology

All Conditions

Neoplasms

Rare Diseases

Recurrence

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Anti-Infective Agents

Amino Acids

Bortezomib

Eflornithine

Antiprotozoal Agents

Antiparasitic Agents

Enzyme Inhibitors

Ornithine

Subjects will take DFMO by mouth 2 times a day for each day of a 21 day cycle and Bortezomib will be given by IV push on days 1, 4, and 8 of each 21 day cycle.

DFMO

Kathleen Neville, MD

The purpose of this research study is to evaluate an investigational drug (DFMO) in combination with bortezomib, for relapsed and refractory neuroblastoma. DFMO is an investigational drug because it has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the safety and tolerability of DFMO in combination with bortezomib as well as the tumors response to this study drug.

Study of Difluoromethylornithine (DFMO) in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma

A Phase I/II Trial of DFMO in Combination With Bortezomib in Patients With Relapsed or Refractory Neuroblastoma

Phase I- To determine the safety and tolerability of DFMO in combination with bortezomib at 3 dose levels of DFMO: 1500mg/m2 twice daily, 2000mg/m2 twice daily, and 2500mg/m2 twice daily in subjects with relapsed or refractory neuroblastoma who receive one full cycle of this dose.

Phase II- Study did not enroll to Phase II. Study completed at end of Phase I.

To determine the overall response rate (ORR) by the presence of radiologically assessable disease by cross-sectional imaging and in MIBG (meta-iodobenzylguanidine) or PET (positron emission computed tomography) scans. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease (PD), At least a 20% increase in the sum of the disease measurements for measurable lesions, Stable Disease, Neither sufficient decrease to qualify for PR or sufficient increase to qualify for PD from study entry. Overall Response (OR) = CR + PR.

Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as at least a 20% increase in the sum of the disease measurements for measurable lesions, taking as reference the smallest disease measurement recorded since the start of treatment (nadir), and minimum 5 mm increase over the nadir or the appearance of one or more new lesions or appearance of positive bone marrow.

Beat NB Cancer Foundation

Because of Ezra

K C Pharmaceuticals Inc.

Giselle Sholler

Beat Childhood Cancer Chair

Subjects that received Dose level 1 - 1500 mg/m2 BID

Phase I: DFMO 1500 mg/m^2

Subjects that received Dose level 2 - 2000 mg/m2 BID

Phase I: DFMO 2000 mg/m^2

Subjects that received Dose level 3 - 2500 mg/m2 BID

Phase I: DFMO 2500 mg/m^2

Phase I: DFMO 2000 mg/m^2"

Phase I: DFMO 2500 mg/m^2"

The highest tolerated DFMO dose from the Phase I dose escalation will be used for the Phase II portion of the study. Subjects will receive oral DFMO twice daily on each day of this 21-day cycle along with Bortezomib and Etoposide.

Phase 2

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

All subjects that took at least one dose of study drug

Giselle Sholler, MD

All subjects that took at least one dose of DFMO

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

Subjects that received Dose level 1 - 1500 mg/m2 BID (twice a day)

Analyzed population only includes evaluable subjects, defined as subjects that made it to an evaluation time point (disease evaluation scans), or had a documented progression of disease prior to evaluation time point.

Determine the Overall Response Rate (ORR) of Participants Using RECIST Criteria

Determine the Progression Free Survival (PFS) of Participants Using Days Until Progression

Phase I: Difluoromethylornithine (DFMO) 1500 mg/m^2

Overall Study

Spots Global Cancer Trial Database for Study of Difluoromethylornithine (DFMO) in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

Useful links and downloads for this trial