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Neuroblastoma Maintenance Therapy Trial (NMTT)

Title NMTT: Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Therapeutic Area Neuroblastoma (pediatric)
Principal Investigator Tishi Shah, MD
Min Age 0 Years
Max Age 30 Years
Gender Both
Contact Michael Hyon
More Information


The purpose of this research study is to evaluate an investigational drug (DFMO) for Neuroblastoma that is in remission. Remission means that there are no current signs of active cancer. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to keep neuroblastoma in remission and will also look at the safety and tolerability of DFMO.

Study Details

Inclusion Criteria

  • Diagnosed with high risk neuroblastoma
  • Currently in complete remission
  • Otherwise normal organ function

Exclusion Criteria

  • No pregnant or nursing females
  • No active infections
  • Not currently receiving cancer treatments

Study Requirements

Participants will need to have exams, tests, and procedures to find out if they can be in the study. This is called “screening.” These exams, tests and procedures are part of regular cancer care and may be done even if the participant does not join the study. If a person is able and willing to participate in this study after the screening is complete, they will take the study drug, DFMO, every day for up to 2 years. Participants may stop the study drug earlier than that if their cancer comes back, they have bad side effects on the study drug, or they decide they want to stop. DFMO is taken by mouth twice a day. It can be swallowed whole or crushed up in food. On day 1, day 91, and day 181 of study drug, extra blood will be taken from participants at 4 times to measure the amount of drug in their system, called pharmacokinetic tests (total of 1 ½ teaspoons or 8 mL). If participants consent, pharmacokinetic tests will be done on day 2 as well. If participants consent, extra blood will be collected and stored for future research on childhood cancer. About 1 ½ teaspoons or 8 mL of extra blood will be collected at screening and every 3 months while the participant is taking study drug. After participants stop taking DFMO, the study doctor will follow up with them to see how they are doing 30 days after the last dose of study drug. After that, the study doctor will follow up with them to see how they are doing every 6 months for the first year and then yearly for 5 years.