Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

  • STATUS
    Recruiting
  • End date
    Oct 1, 2030
  • participants needed
    131
  • sponsor
    Wake Forest University Health Sciences
Updated on 13 May 2022
renal function
cancer
stem cell transplantation
monoclonal antibodies
bone marrow procedure
etoposide
neutrophil count
eflornithine
refractory neuroblastoma
avid
iobenguane
tumor vaccine

Summary

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory neuroblastoma.

Description

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory neuroblastoma.

In this study subjects will receive six 21-day cycles of Etoposide and DFMO followed by an additional 630 days of DFMO alone.

Subjects will be evaluated in 3 arms:

• Arm 1: Subjects who show no active disease after receiving any additional therapy for neuroblastoma that was refractory to standard induction/consolidation therapy.

Refractory: Subjects with progressive disease on upfront therapy OR did not have at least PR on induction OR required additional second line therapy to achieve remission who are now in first remission.

  • Arm 2: Subjects who have previously relapsed and currently show no active disease (in CR2 or greater).
  • Arm 3: Subjects who are relapsed or refractory with active disease.

Details
Condition Neuroblastoma
Treatment Eflornithine
Clinical Study IdentifierNCT04301843
SponsorWake Forest University Health Sciences
Last Modified on13 May 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

All patients must have a pathologically confirmed diagnosis of neuroblastoma, ≤ 30.99 years of age with history of relapsed/refractory neuroblastoma
All patients must have completed upfront therapy with at least 4 cycles of aggressive multi-drug chemotherapy
Specific Criteria by Arm
Arms 1 and 2
Subjects with no active disease
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)
o Note: Patients with residual masses detected by CT/MRI may be considered in CR if their
ii. No evidence of disease metastatic to bone marrow
MIBG is negative or if MIBG positive and evaluated by PET and found to have negative PET
Arm 3
scans; biopsy confirmation may be considered if there is still reasonable concern for
Measurable or evaluable disease, including at least one of the following
persistent disease but is not required
Measurable tumor by CT or MRI; or a positive MIBG and PET; or positive bone marrow
biopsy/aspirate in at least one site
Timing from prior therapy: Enrollment (first dose of DFMO) no later than 60 days from
last dose of the most recent therapy
Subjects must have fully recovered from the acute toxic effects of all prior anti-
cancer chemotherapy and be within the following timelines
Myelosuppressive chemotherapy: Must not have received within 2 weeks of
enrollment onto this study (6 weeks if prior nitrosourea)
Stem Cell Transplant
Hematopoietic growth factors: At least 5 days since the completion of therapy
Allogeneic: No evidence of active graft vs. host disease
with a growth factor
Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy
MIBG Therapy: At least 8 weeks since treatment with MIBG therapy
with a biologic agent. For agents that have known adverse events occurring beyond
Subjects must have a Lansky or Karnofsky Performance Scale score of 60% or higher
days after administration, this period must be extended beyond the time during
Life expectancy > 2 months
which adverse events are known to occur. The duration of this interval must be
discussed with the Study Chair
Subjects must have adequate organ functions at the time of registration
Immunotherapy: At least 6 weeks since the completion of any type of
immunotherapy, e.g. tumor vaccines, CAR-T cells
Anti-GD2 Monoclonal antibodies: At least 2 weeks must have elapsed since prior
treatment with a monoclonal antibody
XRT: At least 14 days since the last treatment except for radiation delivered
with palliative intent to a non-target site
Allo/Auto: ≥ 2 months must have elapsed since transplant
All clinical and laboratory studies for organ functions to determine eligibility must
be performed within 7 days prior to first dose of study drug unless otherwise
indicated below
Hematological: Total absolute neutrophil count ANC ≥750/μL
Liver: Subjects must have adequate liver function as defined by AST and ALT <5x
upper limit of normal (Normal=45), Bilirubin <1.5x upper limit normal
(Normal=1.0). Normal PT, PTT, fibrinogen
Renal: Adequate renal function defined as (perform one of the following)
Creatinine clearance or radioisotope GFR 70 mL/min/1.73 m2 or greater or a serum
creatinine based on age/gender
Females of childbearing potential must have a negative pregnancy test. Patients of
childbearing potential must agree to use an effective birth control method. Female
patients who are lactating must agree to stop breast-feeding
Written informed consent in accordance with institutional and FDA guidelines must be
obtained from all subjects (or patients' legal representative)

Exclusion Criteria

BSA of <0.25 m2
Subjects that received a dose of DFMO in combination with etoposide are not eligible
Subjects that received DFMO at a dose higher than 1000mg/m2 BID prior to this study
are not eligible
Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation
Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
not eligible. Subjects must have fully recovered from hematological and bone marrow
suppression effects of prior chemotherapy
Infection: Subjects who have an uncontrolled infection are not eligible until the
infection is judged to be well controlled in the opinion of the investigator
Subjects who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study, or in whom compliance is likely to be
suboptimal, should be excluded
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