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Anti-cancer drug

DFMO + Etoposide for Neuroblastoma

Phase 2
Recruiting
Research Sponsored by Giselle SaulnierSholler
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
i. No evidence of residual disease by CT/MRI and MIBG scan (or PET for patients who have a history of MIBG non-avid disease).
Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the Study Chair.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years plus 5 years follow up
Awards & highlights

Study Summary

This trial will test the combination of DFMO and etoposide for subjects with relapsed/refractory neuroblastoma.

Who is the study for?
This trial is for individuals under 31 years old with neuroblastoma that has come back or didn't respond to treatment. They must have completed at least 4 cycles of intense chemotherapy, have good organ function, and a performance score of 60% or higher. Women who can have children need a negative pregnancy test and agree to use birth control.Check my eligibility
What is being tested?
The study tests Eflornithine (DFMO) in combination with etoposide on patients with relapsed/refractory neuroblastoma. It's an open-label, multicenter trial meaning all participants know what treatment they're getting and it involves multiple locations.See study design
What are the potential side effects?
Possible side effects include suppression of bone marrow leading to low blood counts, digestive issues from etoposide such as nausea or vomiting, potential liver problems indicated by changes in certain blood tests, fatigue, hair loss, and allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My scans show no remaining cancer.
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It's been over a week since my last biologic treatment.
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My scans show no active cancer, even if there are leftover masses.
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I haven't had chemotherapy that lowers blood cell counts in the last 2 weeks (or 6 weeks for a specific type).
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I have recovered from previous cancer treatments without lingering side effects.
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My liver functions are within the required limits.
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My cancer has not spread to my bone marrow.
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I have completed at least 4 cycles of intense chemotherapy.
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I do not have active symptoms from a transplant rejection.
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It has been over 8 weeks since my last MIBG therapy.
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It has been over 2 months since my transplant.
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I can do most activities but may need help.
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My cancer can be seen on scans or confirmed through a biopsy.
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I am 30 years old or younger with a confirmed neuroblastoma diagnosis that has come back or didn't respond to treatment.
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My liver functions are within the required range.
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My kidney function is good based on tests.
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I am 30 years old or younger with a confirmed diagnosis of neuroblastoma that has come back or did not respond to treatment.
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My scans show no signs of cancer remaining.
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It's been over 2 weeks since my last monoclonal antibody treatment.
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It's been over 2 weeks since my last radiation therapy, except for palliative care.
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My condition is currently stable with no active disease.
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I have completed at least 4 cycles of intense chemotherapy.
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My cancer has not spread to my bone marrow.
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My cancer can be seen on scans or confirmed through a biopsy.
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It has been over 2 months since my transplant.
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I have undergone a stem cell transplant.
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It's been over 5 days since I last received treatment with growth factors.
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I do not have active symptoms from a transplant rejection.
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My kidney function is good based on tests.
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I can do most activities but may need help.
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It has been over 8 weeks since my last MIBG therapy.
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My organs are functioning well.
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It has been over 6 weeks since I last received any form of immunotherapy.
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I am eligible for either the first or second part of the study.
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I have given my written consent to participate.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years plus 5 years follow up
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years plus 5 years follow up for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with event free survival (EFS) during study
Secondary outcome measures
Determine the Overall Response Rate (ORR) of Participants using INSS Response Evaluation Criteria.
Length of time that participants experience Overall Survival (OS)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Side effects data

From 2019 Phase 3 trial • 171 Patients • NCT01483144
21%
nausea
14%
upper respiratory tract infection
14%
abdominal pain
14%
headache
13%
abdominal pain upper
13%
gastroenteritis
13%
diarrhoea
13%
rectal haemorrhage
11%
vomiting
11%
rash
11%
haematochezia
11%
nasopharyngitis
9%
flatulence
9%
influenza like illness
9%
back pain
9%
oropharyngeal pain
7%
arthralgia
7%
fatigue
7%
myalgia
7%
influenza
7%
dizziness
7%
sinusitis
5%
gastroenteritis viral
5%
cough
5%
pouchitis
5%
constipation
5%
gastritis erosive
5%
pruritus
5%
dry skin
5%
small bowel obstruction
5%
neck pain
5%
urticaria
4%
abdominal distension
4%
weight increased
4%
small intestinal obstruction
4%
dyspepsia
4%
anxiety
4%
decreased appetite
4%
alopecia
4%
urinary tract infection
2%
nasal congestion
2%
bursitis
2%
seasonal allergy
2%
pancreatitis acute
2%
seroma
2%
wound dehiscence
2%
hyperglycaemia
2%
bronchitis
2%
contusion
2%
inguinal hernia
2%
nephritis
2%
renal failure acute
2%
depression
2%
ileus
2%
lung adenocarcinoma
2%
insomnia
2%
ear pain
2%
pulmonary mass
2%
psychotic disorder
100%
80%
60%
40%
20%
0%
Study treatment Arm
Eflornithine Plus Sulindac
Eflornithine Plus Sulindac Placebo
Sulindac Plus Eflornithine Placebo

Trial Design

1Treatment groups
Experimental Treatment
Group I: Eflornithine (DFMO)Experimental Treatment1 Intervention
In this study subjects will receive six 21-day cycles of Etoposide and DFMO followed by an additional 630 days of DFMO alone. Etoposide will be given at 50 mg/m2/dose PO daily for the first 14 days of each 21 days until 6 cycles of etoposide are completed. DFMO (difluoromethylornithine) will be given at a dose of 1000 mg/m2 BID on each day of study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eflornithine
1998
Completed Phase 3
~830

Find a Location

Who is running the clinical trial?

Giselle SaulnierShollerLead Sponsor
21 Previous Clinical Trials
2,085 Total Patients Enrolled
18 Trials studying Neuroblastoma
1,458 Patients Enrolled for Neuroblastoma
Wake Forest University Health SciencesLead Sponsor
1,234 Previous Clinical Trials
1,001,926 Total Patients Enrolled
19 Trials studying Neuroblastoma
1,515 Patients Enrolled for Neuroblastoma
Beat NB Cancer FoundationOTHER
5 Previous Clinical Trials
1,003 Total Patients Enrolled
5 Trials studying Neuroblastoma
1,003 Patients Enrolled for Neuroblastoma

Media Library

Eflornithine (Anti-cancer drug) Clinical Trial Eligibility Overview. Trial Name: NCT04301843 — Phase 2
Neuroblastoma Research Study Groups: Eflornithine (DFMO)
Neuroblastoma Clinical Trial 2023: Eflornithine Highlights & Side Effects. Trial Name: NCT04301843 — Phase 2
Eflornithine (Anti-cancer drug) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04301843 — Phase 2

Frequently Asked Questions

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

Is this a pioneering trial for its kind?

"Presently, 11 live clinical trials are taking place for Eflornithine across 699 cities and 12 countries. Cancer Prevention Pharmaceuticals, Inc. initiated the first trial in 2013 with 1340 participants which has since gone through its Phase 3 drug approval stage. In addition to this initial study, 10 other experiments have also been concluded so far."

Answered by AI

What is the aggregate number of participants engaging in this research project?

"This clinical trial necessitates 131 participants who meet the inclusion standards. Patients can join in from sites such as Augusta University Health (Augusta, Georgia) and Children's Medical Center (Dallas, Texas)."

Answered by AI

Are there still opportunities to join this research endeavor?

"Affirmative. The information posted on clinicaltrials.gov affirms that this medical trial is actively recruiting 131 patients at 23 sites, beginning September 25th 2020 and most recently updated April 20th 2022."

Answered by AI

How widely dispersed is the experimental program?

"Augusta University Health in Augusta, Georgia, Children's Medical Center in Dallas, Texas, and the Medical University of South carolina located in Charleston are some of the 23 research hubs associated with this medical trial."

Answered by AI

What prior investigations have been conducted regarding Eflornithine?

"In 2013, the first studies of eflornithine were conducted at Lake Norman Hematology Oncology Specialists-Mooresville. To date, 10 trials have been concluded and there are 11 ongoing clinical tests - primarily located in Augusta, Georgia."

Answered by AI

To what extent does Eflornithine pose a risk to individuals?

"Although there is some evidence of Eflornithine's safety, the lack of data concerning efficacy gives this medication a 2 on our team's safety scale."

Answered by AI
~74 spots leftby Oct 2028