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An Intermediate Expanded Use Trial of DFMO

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03581240
Expanded Access Status : Available
First Posted : July 10, 2018
Last Update Posted : September 28, 2023
Sponsor:
Collaborators:
K C Pharmaceuticals Inc.
USWM, LLC
Information provided by (Responsible Party):
Giselle Sholler, Milton S. Hershey Medical Center

Study Description
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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.

Condition or disease Intervention/treatment
Neuroblastoma Medulloblastoma Typical Teratoid Rhabdoid Tumor Embryonal Tumor With Abundant Neuropil and True Rosettes Ependymoblastoma Medulloepithelioma Drug: eflornithine HCl

Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population, Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: An Intermediate Expanded Use Trial of DFMO

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • 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.

    Other Name: DFMO
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   0 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03581240


Contacts
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Contact: Genevieve Bergendahl, MSN 7175310003 gbergendahl@pennstatehealth.psu.edu

Locations
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United States, Pennsylvania
Penn State Milton S. Hershey Medical Center and Children's Hospital Available
Hershey, Pennsylvania, United States, 17033
Contact: Suzanne Treadway       streadway@hmc.psu.edu   
Principal Investigator: Valerie Brown, MD         
Sponsors and Collaborators
Giselle Sholler
K C Pharmaceuticals Inc.
USWM, LLC
Investigators
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Study Chair: Giselle Sholler, MD Beat Childhood Cancer
More Information
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Additional Information:

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Responsible Party: Giselle Sholler, Study Chair, Milton S. Hershey Medical Center
ClinicalTrials.gov Identifier: NCT03581240    
Other Study ID Numbers: NMTRC006
First Posted: July 10, 2018    Key Record Dates
Last Update Posted: September 28, 2023
Last Verified: September 2023
Additional relevant MeSH terms:
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Neuroblastoma
Medulloblastoma
Rhabdoid Tumor
Neuroectodermal Tumors, Primitive
Neoplasms
Neuroectodermal Tumors, Primitive, Peripheral
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
 Glioma
Neoplasms, Complex and Mixed
Eflornithine
Antineoplastic Agents
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Ornithine Decarboxylase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action