A Study of Reduced Therapy for Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma

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Age: 3 to 21 years old
Gender:
Any
Healthy Volunteers: No
Keywords: Medulloblastoma, Average-Risk WNT-Driven Medulloblastoma
Type: Drug study, Phase 2
Target:
5 Participants
Investigator:
Description
You are being asked to take part in this research study because you have been diagnosed with a cancer called medulloblastoma. This is a type of childhood brain tumor.

This cancer is called “average-risk” or “Classic Medulloblastoma” because:
- you are 3 years of age or older
- the tumor is in the very back of the brain in an area called the posterior fossa
- all or almost all of the tumor was removed by surgery
- the cancer has not spread to other parts of your brain or spinal cord.

The term, risk, refers to the chance of the cancer coming back after treatment.

There are four main types of Medulloblastoma. They are set apart by the presence, absence, or changing levels of certain proteins and other molecules in the tumor cells. Some of these different types of cancer respond differently to current treatments.

This study if for patients whose doctors think they have Classic Medulloblastoma of the WNT sub-type type. WNT-subtype medulloblastoma patients have had better survival than other types of medulloblastoma. WNT-subtype medulloblastoma can be found using a number of specialized molecular tests.

People with WNT-subtype medulloblastoma will get less therapy than other types of medulloblastoma. So, we want to make sure that the diagnosis is correct before you are given any treatment on this research study. It is unknown whether less treatment will result in no change in the chance of cure or a decreased chance of a cure.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  • Patients must be newly diagnosed and have a confirmed molecular diagnosis of classical histologic type (non large cell/anaplastic [LC/A]) WNT medulloblastoma from rapid central pathology screening review on APEC14B1 (immunohistochemistry [IHC]/molecular screening [positive nuclear beta (B)-catenin by IHC and positive for catenin beta 1 [CTNNB1] mutation) and confirmation of =< 1.5 cm^2 maximal cross-sectional area of residual tumor from rapid central imaging review
  • Patient must have negative lumbar cerebrospinal fluid (CSF) cytology; CSF cytology for staging should be performed preferably no sooner than 14 days post operatively to avoid false positive CSF; ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study
  • Note: patients with positive CSF cytology obtained prior to 14 days after surgery may have cytology repeated to determine eligibility and final CSF status
  • Patients must have eligibility confirmed by rapid central imaging review on APEC14B1; standard whole brain magnetic resonance imaging (MRI) with and without contrast (gadolinium) and spine MRI with contrast (gadolinium) must be performed at the following time points:
  • Pre-operative to include an MRI of the brain with and without contrast (including post-contrast three-dimensional [3D] T1-weighted image [T1WI] and post-contrast fluid-attenuated inversion recovery [FLAIR])
  • Pre-operative spinal MRI with gadolinium; post-operative staging spinal MRI may be obtained if pre-operative imaging is not possible or is suboptimal; pre-operative spine imaging is strongly preferred, due to the potential of post-operative sequelae, which could affect metastasis detection
  • Post-operative brain MRI within 72 hours of surgery
  • Patients must be enrolled on ALTE07C1 prior to enrollment on ACNS1422
  • Patients must be enrolled within 36 days of definitive diagnostic surgery (day 0)
  • Note: patients must begin treatment within 36 days of definitive surgery
  • Patients must have no previous radiotherapy or chemotherapy other than corticosteroids
  • Peripheral absolute neutrophil count (ANC) >= 1000/uL
  • Platelet count >= 100,000/uL (transfusion independent)
  • Hemoglobin >= 10.0 g/dL (may receive red blood cell [RBC] transfusions)
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
  • 3 to < 6 years of age: maximum (max) serum creatinine 0.8 mg/dL (males and females)
  • 6 to < 10 years of age: max serum creatinine 1 mg/dL (males and females)
  • 10 to < 13 years of age: max serum creatinine 1.2 mg/dL (males and females)
  • 13 to < 16 years of age: max serum creatinine 1.5 md/dL (males) and 1.4 md/dL (females)
  • = 16 years of age: max serum creatinine 1.7 mg/dL (males) and 1.4 mg/dL (females)

  • The threshold creatinine values were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control and Prevention (CDC)
  • Total or direct bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
  • Serum glutamate pyruvate (SGPT) (alanine aminotransferase [ALT]) =< 110 U/L (for the purpose of this study, the upper limit of normal [ULN] for SGPT is 45 U/L)
  • Central nervous system function defined as:
  • Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
  • Patients must not be in status epilepticus, a coma or on assisted ventilation at the time of study enrollment
  • All patients and/or their parents or legal guardians must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion criteria:

  • Patients with metastatic disease by either MRI evaluation (brain and spine) or lumbar CSF cytology are not eligible; patients who are unable to undergo a lumbar puncture for assessment of CSF cytology are ineligible
  • Patients must not have received any prior tumor-directed therapy other than surgical intervention and corticosteroids
  • Female patients who are pregnant are ineligible
  • Lactating females are not eligible unless they have agreed not to breastfeed their infants
  • Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
  • Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
You will receive treatment on this study for about one year. After treatment, you will have follow-up examinations and medical tests. We would like to continue to follow your health every year for about 10 years after you enter this study. This will help us better understand the long-term effects of the study treatments.
Recruitment period
From March 7, 2018
Location
UC Davis Comprehensive Cancer Center
4501 X Street
Sacramento, CA 95817
Research Topic
Conditions:
  • Medulloblastoma
  • Untreated Childhood Medulloblastoma

Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.


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