Proteasome Inhibitor

DFMO for Neuroblastoma

Arkansas Children's Hospital, Little Rock, AR

DFMO +1 morePhase 1 & 2Waitlist AvailableResearch Sponsored by Wake Forest University Health Sciences

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Must not have

Be older than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow ups3 years

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Study Summary

This trial is testing an investigational drug (DFMO) in combination with bortezomib for relapsed and refractory neuroblastoma. It will evaluate safety, tolerability, and tumor response.

Eligible Conditions

Neuroblastoma

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow ups ~ 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow ups ~ 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Determine the Overall Response Rate (ORR) of Participants using RECIST criteria

Secondary outcome measures

Biology studies

Correlate PET scan changes with Progression Free Survival

Correlate urinary polyamine levels with response and progression of disease in neuroblastoma.

+2 more

Side effects data

From 2015 Phase 1 trial • 21 Patients • NCT01059071

24%

Neutorpenia

19%

Anemia

14%

AST elevation

10%

Progression of Disease resulting in death

10%

ALT elevation

10%

Constipation

Rash

Neuropathy

Pain

Sleep disturbance

urinary retention

vomiting

Conjunctivitis

Thrombocytopenia

Leukopenia

Anorexia

Diarrhea

GGT elevation

Hypoalbuminemia

Hypophosphatemia

Infection, sinus

Mouth pain

Nausea

100%

80%

60%

40%

20%

Study treatment Arm

DFMO and Etoposide

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

1Treatment groups

Experimental Treatment

Group I: DFMO and BortezomibExperimental Treatment2 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bortezomib

FDA approved

Eflornithine

FDA approved

Do I qualify?Apply below to find out

Find a site

Who is running the clinical trial?

Wake Forest University Health SciencesLead Sponsor

1,195 Previous Clinical Trials

999,115 Total Patients Enrolled

19 Trials studying Neuroblastoma

1,630 Patients Enrolled for Neuroblastoma

Beat NB Cancer FoundationOTHER

5 Previous Clinical Trials

1,118 Total Patients Enrolled

5 Trials studying Neuroblastoma

1,118 Patients Enrolled for Neuroblastoma

Because of EzraOTHER

1 Previous Clinical Trials

140 Total Patients Enrolled

1 Trials studying Neuroblastoma

140 Patients Enrolled for Neuroblastoma

K C Pharmaceuticals Inc.Industry Sponsor

4 Previous Clinical Trials

771 Total Patients Enrolled

4 Trials studying Neuroblastoma

771 Patients Enrolled for Neuroblastoma

Giselle SaulnierShollerLead Sponsor

21 Previous Clinical Trials

2,200 Total Patients Enrolled

18 Trials studying Neuroblastoma

1,573 Patients Enrolled for Neuroblastoma

Kathleen Neville, MDStudy Chair

Children's Mercy Hospital Kansas City

Media Library

Bortezomib (Proteasome Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02139397 — Phase 1 & 2

Neuroblastoma Research Study Groups: DFMO and Bortezomib

Neuroblastoma Clinical Trial 2023: Bortezomib Highlights & Side Effects. Trial Name: NCT02139397 — Phase 1 & 2

Bortezomib (Proteasome Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02139397 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could you compare and contrast this DFMO study to others like it?

"DFMO's first clinical trial was completed in 2004 at the National Institutes of Health Clinical Center. As of now, 395 have been completed with 156 more underway. A significant amount of these ongoing trials are based out of Little Rock, Arkansas."

Answered by AI

Are we looking for more participants in this experiment?

"The clinical trial in question is not recruiting new participants, as per the information on clinicaltrials.gov. This study was first posted on May 1st, 2014 and was last updated on April 20th, 2022. 321 other trials are currently enrolling patients."

Answered by AI

What are the benefits of using DFMO?

"DFMO has been successful in treating hair removal therapy, meningoencephalitic stage trypanosoma brucei gambiense infection, and multiple myeloma."

Answered by AI

How many different sites are conducting this research?

"There are a total of six different hospitals participating in this study. These include the Arkansas Children's Hospital in Little Rock, Medical University of South Carolina in Charleston, and Arnold Palmer Hospital for Children- MD Anderson in Orlando."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

Giselle SaulnierSholler 2014. "Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02139397.