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
Sponsored by
About this trial
This is an expanded access trial for Neuroblastoma
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.
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
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
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
Email
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
Email
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
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An Intermediate Expanded Use Trial of DFMO
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