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An Intermediate Expanded Use Trial of DFMO

Primary Purpose

Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor

Status

Available

Phase

Locations

United States

Study Type

Expanded Access

Intervention

eflornithine HCl

About this trial

This is an expanded access trial for Neuroblastoma

Eligibility Criteria

0 Years - 30 Years (Child, Adult)All Sexes

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.

Sites / Locations

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

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT03581240

First Posted

June 26, 2018

Last Updated

September 26, 2023

Sponsor

Giselle Sholler

Collaborators

K C Pharmaceuticals Inc., USWM, LLC

1. Study Identification

Unique Protocol Identification Number

NCT03581240

Brief Title

An Intermediate Expanded Use Trial of DFMO

Official Title

An Intermediate Expanded Use Trial of DFMO

Study Type

Expanded Access

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Available

Study Start Date

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Primary Completion Date

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Study Completion Date

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3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Giselle Sholler

Collaborators

K C Pharmaceuticals Inc., USWM, LLC

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

eflornithine HCl

Other Intervention Name(s)

DFMO

Intervention Description

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.

10. Eligibility

Sex

All

Minimum Age & Unit of Time

0 Years

Maximum Age & Unit of Time

30 Years

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: High risk neuroblastoma patients that have completed standard of care upfront therapy and are not eligible for NMTRC014. Medulloblastoma patients who have completed standard of care therapies. Relapsed/refractory neuroblastoma patients who have completed standard of care therapies. 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Genevieve Bergendahl, MSN

Phone

7175310003

gbergendahl@pennstatehealth.psu.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Giselle Sholler, MD

Organizational Affiliation

Beat Childhood Cancer

Official's Role

Study Chair

Facility Information:

Facility Name

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

City

Hershey

State/Province

Pennsylvania

ZIP/Postal Code

17033

Country

United States

Individual Site Status

Available

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Suzanne Treadway

streadway@hmc.psu.edu

First Name & Middle Initial & Last Name & Degree

Valerie Brown, MD

12. IPD Sharing Statement

Links:

URL

http://beatcc.org

Description

Beat Childhood Cancer website

Learn more about this trial

An Intermediate Expanded Use Trial of DFMO

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