Official Title
An Intermediate Expanded Use Trial of DFMO
Brief Summary

To provide DFMO in an expanded use setting to subjects with relapsed rare tumors withincreased LIN28 expression or MYCN amplification or up regulation of ornithinedecarboxylase.

Available
Intermediate-size Population
Treatment IND/Protocol
Neuroblastoma
Medulloblastoma
Typical Teratoid Rhabdoid Tumor
Embryonal Tumor With Abundant Neuropil and True Rosettes
Ependymoblastoma
Medulloepithelioma

Drug: eflornithine HCl

In this study subjects without CNS disease will receive oral difluoromethylornithine
(DFMO) at a dose of 500 to 1000 mg/m2 BID

Subjects with CNS disease will receive a dose of 2500 mg/m2/dose BID in order to
facilitate crossing into the CNS.
Other Name: DFMO

Eligibility Criteria

Inclusion Criteria:

- Age: 0-30 years at the time of initial diagnosis.

- Diagnosis: Histologic verification at either the time of original diagnosis or a
previous relapse of High Risk neuroblastoma, medulloblastoma, atypical teratoid
rhabdoid tumor, embryonal tumor with abundant neuropil and true rosettes,
ependymoblastoma, medulloepithelioma and other rare pediatric MYC, ODC or LIN28/Let7
driven tumors (each type will form a new subset).

- Disease Status: Subjects must be in one of the following disease categories:

1. High risk neuroblastoma patients that have completed standard of care upfront
therapy and are not eligible for NMTRC014.

2. Medulloblastoma patients who have completed standard of care therapies.

3. Relapsed/refractory neuroblastoma patients who have completed standard of care
therapies.

4. Rare tumors with increased LIN28 expression or MYCN amplification or up
regulation of ornithine decarboxylase who have completed standard of care
therapies.

- Subjects are not eligible to enroll on DFMO studies NMTRC002, NMTRC003, NMTRC010, or
NMTRC014.

- A negative serum or urine pregnancy test is required for female subjects of child
bearing potential (onset of menses or ≥13 years of age).

- Both male and female post-pubertal study subjects need to agree to use one of the
more effective birth control methods during treatment and for six months after
treatment is stopped. These methods include total abstinence (no sex), oral
contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants
(Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one
of these cannot be used, contraceptive foam with a condom is recommended.

- Informed Consent: All subjects and/or legal guardians must sign informed written
consent. Assent, when appropriate, will be obtained according to institutional
guidelines

Exclusion Criteria:

- BSA (m2) of <0.25

- Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation.

Eligibility Gender
All
Eligibility Age
Minimum: 0 Years ~ Maximum: 30 Years
Countries
United States
Locations

Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States

Investigator: Suzanne Treadway
streadway@hmc.psu.edu

Investigator: Valerie Brown, MD

Contacts

Giselle Sholler, MD, Study Chair
Beat Childhood Cancer

Giselle Sholler
NCT Number
MeSH Terms
Neuroblastoma
Medulloblastoma
Rhabdoid Tumor
Neuroectodermal Tumors, Primitive
Eflornithine