An Intermediate Expanded Use Trial of DFMO

NCT ID: NCT03581240

Last Updated: 2025-08-07

Basic Information

• Recruitment Status: AVAILABLE
• Study Classification: EXPANDED_ACCESS

Brief Summary

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

Conditions

Neuroblastoma; Medulloblastoma; Typical Teratoid Rhabdoid Tumor; Embryonal Tumor With Abundant Neuropil and True Rosettes; Ependymoblastoma; Medulloepithelioma

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

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.

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

K C Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: collaborator

USWM, LLC

UNKNOWN

Sponsor Role: collaborator

Giselle Sholler

OTHER

Sponsor Role: lead

Responsible Party

Giselle Sholler

Study Chair

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Giselle Sholler, MD

Role: STUDY_CHAIR

Beat Childhood Cancer

Locations

Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, United States

Site Status: AVAILABLE

Countries

United States

Central Contacts

BCC Enroll

Role: CONTACT

BCCEnroll@pennstatehealth.psu.edu

7175310003

Facility Contacts

Suzanne Treadway

Role: primary

streadway@hmc.psu.edu

Related Links

http://beatcc.org

Beat Childhood Cancer website

Other Identifiers

NMTRC006

Identifier Type: -

Identifier Source: org_study_id