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An Intermediate Expanded Use Trial of DFMO

NCT03581240 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2026-02-20

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • K C Pharmaceuticals Inc.

    collaborator INDUSTRY

  • USWM, LLC

    collaborator UNKNOWN

  • Giselle Sholler

    lead OTHER

Principal Investigators

  • Giselle Sholler, MD · Beat Childhood Cancer

Eligibility

Min Age
0 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03581240 on ClinicalTrials.gov