BridgeMD
New treatment option for Medulloblastoma
Official title DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
ClinicalTrials.gov ID: NCT04696029
What this study is testing
What is Difluoromethylornithine?
Difluoromethylornithine is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for medulloblastoma.
Also referred to as Eflornithine, DFMO.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 21
You may be able to join if
- Age: 0-21 years of age at diagnosis
- Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either...
- Metastatic non-MYC amplified Group 3
- Metastatic Group 4
- Metastatic non-WNT/non-SHH (Must be non-MYC amplified) Cohort 2- Molecular Very High Risk
You likely can't join if
- BSA of \<0.25 m2
- Metastatic disease outside of CNS
- Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
- Investigational Drugs: people who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: people who are currently receiving other anticancer agents are not eligible. people must have fully recovered from the...
- Infection: people who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the...
See the full eligibility criteria
- Age: 0-21 years of age at diagnosis
- Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling. Cohort 1- Molecular High Risk:
- Metastatic non-MYC amplified Group 3
- Metastatic Group 4
- Metastatic non-WNT/non-SHH (Must be non-MYC amplified) Cohort 2- Molecular Very High Risk
- Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
- MYC amplified Group 3
- Non-WNT, non-SHH infant (\< 3 yrs) Cohort 3: Relapsed/Refractory Medulloblastoma
- Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
- Tumor imaging studies including: Brain and spine MRI
- Lumbar Puncture only if previously positive
- Bone Marrow aspiration/biopsy only if previously positive
- This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
- Disease Status: people must have no evidence of disease, or stable\ residual nonbulky\ \ disease. \ Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart \ \ Non-bulky...
- Timing from prior therapy: Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell...
- Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
- All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
- Patients must have adequate organ functions at the time of registration:
- Hematological: Hematological recovery as defined by ANC ≥750/μL, platelets ≥30 (non-transfused x 7 days)
- Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
- Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or a serum creatinine based on age/gender
- Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop...
- Written informed consent in accordance with institutional and FDA guidelines must be obtained from all people (or patients' legal representative).
- BSA of \<0.25 m2
- Metastatic disease outside of CNS
- Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
- Investigational Drugs: people who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: people who are currently receiving other anticancer agents are not eligible. people must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
- Infection: people who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
- people who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Oakland, California, United States
- San Diego, California, United States
- Stanford, California, United States
- Hartford, Connecticut, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- St. Petersburg, Florida, United States
- Tampa, Florida, United States
- Honolulu, Hawaii, United States
- Chicago, Illinois, United States
- Lexington, Kentucky, United States
- Louisville, Kentucky, United States
- Kansas City, Missouri, United States
- St Louis, Missouri, United States
- Hackensack, New Jersey, United States
- Charlotte, North Carolina, United States
- Oklahoma City, Oklahoma, United States
- Portland, Oregon, United States
- Hershey, Pennsylvania, United States
+ 4 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Oakland, California, United States; San Diego, California, United States; Stanford, California, United States; Hartford, Connecticut, United States; Miami, Florida, United States and 18 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.