Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma
|ClinicalTrials.gov Identifier: NCT02139397|
Recruitment Status : Active, not recruiting
First Posted : May 15, 2014
Last Update Posted : November 18, 2020
|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma Recurrent||Drug: DFMO Drug: Bortezomib||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/II Trial of DFMO in Combination With Bortezomib in Patients With Relapsed or Refractory Neuroblastoma|
|Actual Study Start Date :||May 2014|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
Experimental: DFMO and Bortezomib
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.
Other Name: Velcade
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 months ]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.
- Determine the Overall Response Rate (ORR) of Participants using RECIST criteria [ Time Frame: 3 years ]To determine the overall response rate (ORR) by the presence of radiologically assessable disease by cross-sectional imaging and in MIBG or PET scans.
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 3 years ]Phase II- To continue to determine the safety and tolerability of DFMO in combination with bortezomib in subjects with relapsed or refractory neuroblastoma.
- Determine the Progression Free Survival (PFS) of Participants using days until progression [ Time Frame: 3 years ]
To evaluate the activity of DFMO in combination with bortezomib based on:
- Progression free survival (PFS)
- Correlate PET scan changes with Progression Free Survival [ Time Frame: 3 years ]For subjects followed with PET scan: comparison of changes in PET activity and correlation with PFS
- Biology studies [ Time Frame: 3 years ]Urine, blood, bone marrow and tumor biological Correlates will be explored. Dose effect of DFMO on biological correlates will also be explored.
- Correlate urinary polyamine levels with response and progression of disease in neuroblastoma. [ Time Frame: 3 years ]Correlation of urinary polyamine levels with response and progression of disease in neuroblastoma.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02139397
|United States, Arkansas|
|Arkansas Children's Hospital|
|Little Rock, Arkansas, United States, 72202|
|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|
|United States, South Carolina|
|Medical University of South Carolina|
|Charleston, South Carolina, United States, 29425|
|Study Chair:||Kathleen Neville, MD||Children's Mercy Hospital Kansas City|