NeuroblastomaNCT03581240
An Intermediate Expanded Use Trial of DFMO
A study testing eflornithine HCl in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 30 years
- Conditions the study is for
- Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor
- What is being tested?
- eflornithine HCl
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 30 years
- All sexes
- Child, Adult
Who may be able to join
- 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.
Show all 11Show fewer
- 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
Who cannot join
- BSA (m2) of <0.25
- Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
Trial locations and contacts
1 site in 1 country.
United States1 site
- Available
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033
Penn State Clinical Trials, ExtractClinicalTrials@pennstatehealth.psu.edu
Valerie Brown
About this study
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
- Being tested
- eflornithine HCl
- Medical subject headings
- Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor, Neuroectodermal Tumors, Primitive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eflornithine HClDrug
Also called DFMO
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- K C Pharmaceuticals Inc., USWM, LLC
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Giselle Sholler, MD, Study chair, Beat Childhood Cancer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03581240
- Sponsor's protocol ID
- NMTRC006
- Status last verified
- Feb 2026
- First submitted
- 26 Jun 2018
- Last updated
- 20 Feb 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Intermediate Expanded Use Trial of DFMO
- ClinicalTrials.gov ID
- NCT03581240
- Acronym / sponsor ID
- NMTRC006
- Conditions
- Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor, Embryonal Tumor With Abundant Neuropil and True Rosettes, Ependymoblastoma, Medulloepithelioma
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- K C Pharmaceuticals Inc., USWM, LLC
- First posted
- 10 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 0 to 30 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 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:
- 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).
Key exclusion criteria (2 listed)
- BSA (m2) of <0.25
- Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| eflornithine HClAlso: DFMOIn 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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Beat Childhood Cancer websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Giselle Sholler, MD study chair
Beat Childhood Cancer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing eflornithine HCl in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact BCC Enroll (7175310003, BCCEnroll@pennstatehealth.psu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.