Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|