Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma
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ClinicalTrials.gov Identifier: NCT02212574 |
Recruitment Status :
Suspended
First Posted : August 8, 2014
Last Update Posted : January 12, 2021
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Condition or disease | Intervention/treatment | Phase |
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Medulloblastoma | Drug: Lomustine Drug: Vincristine Drug: Cisplatin Drug: Cyclophosphamide Drug: Mesna | Not Applicable |
There will be 9 cycles of chemotherapy. There are two different kinds of cycles given. They are referred to as A and B.
Cycle A lasts for 6 weeks and Cycle B lasts for 4 weeks. B cycles are given after the completion of two A cycles.
Below are the details of the drugs and schedules for A and B cycles.
Cycle A (This cycle lasts 42 days)
- Lomustine (CCNU) is given by mouth on Day 1.
- Vincristine is given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, and 15.
- Cisplatin is given directly into a vein over 8 hours on Day 1
Cycle B (This cycle lasts 28 days)
- Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2.
- MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide and repeated at 3 and 6 hours.
- Vincristine is given directly into a vein directly into the vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 and 8.
You may also get a supportive care drug called a myeloid growth factor (filgrastim or pegfilgrastim). This drug will help your blood counts recover after the chemotherapy is given.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 13 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pilot Study Assessing the Feasibility of a Surgery and Chemotherapy-Only Approach in the Upfront Therapy of Children With Wnt Positive Standard Risk Medulloblastoma |
Actual Study Start Date : | January 7, 2016 |
Estimated Primary Completion Date : | May 2021 |
Estimated Study Completion Date : | May 2022 |

Arm | Intervention/treatment |
---|---|
Chemotherapy
Chemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1. Vincristine is given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, and 15. Cisplatin is given directly into a vein over 8 hours on Day 1. This cycle lasts 42 days. Chemotherapy Cycle B Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2. MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide and repeated at 3 and 6 hours. Vincristine is given directly into a vein directly into the vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 and 8.This cycle lasts 28 days |
Drug: Lomustine
Chemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1.
Other Name: CCNU Drug: Vincristine Chemotherapy Cycle A Vincristine is given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, and 15.
Other Name: Oncovin Drug: Cisplatin Chemotherapy Cycle A Cisplatin is given directly into a vein over 8 hours on Day 1.
Other Name: Cisplatinum Drug: Cyclophosphamide Chemotherapy Cycle B Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2.
Other Name: Cytoxan Drug: Mesna Chemotherapy Cycle B MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide and repeated at 3 and 6 hours.
Other Name: Mesnex Drug: Vincristine Chemotherapy Cycle B Vincristine is given directly into a vein directly into the vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 and 8.
Other Name: Oncovin |
- Progression Free Survival [ Time Frame: 3 years ]To determine the feasibility of treating newly diagnosed children with non-metastatic, standard risk, Wnt positive medulloblastoma with a chemotherapy-only approach. Primary outcome measure of this study will be progression-free survival.
- Patterns of Failure [ Time Frame: 3 years ]To evaluate the patterns of failure in those children that do not have progressive disease, progression free survival and overall survival.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 3 Years to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Participants must have classical histology posterior fossa medulloblastoma as determined by institutional neuro-pathological evaluation.
- Sufficient pathologic material must be available for central analysis and review
- Tumors will be deemed Wnt positive if, at the time of central analysis, there is:
- Monosomy 6 as determined by array CGH
- Gene transcript detection by NanoString supporting Wnt+ medulloblastoma
- Absence of large-cell, anaplastic histology
- Nuclear b-catenin IHC will be determined, but not required for the diagnosis
- Absence of residual or disseminated disease as defined by the following criteria: Minimal residual disease as determined by post-operative imaging preferably performed within 48 hours of resection (and at most 28 days post-surgery), i.e. gross total resection or residual disease of <1.5cm2 on post-operative imaging.
No evidence of metastatic disease in the brain, spine or cerebral spinal fluid (CSF). Assessments must include MRI imaging of the brain and spine with and without contrast and a lumbar puncture for CSF cytology
- Diagnostic imaging (pre and post contrast) must be forwarded to Dana-Farber Cancer Institute (DFCI) for central review to confirm eligibility
- Patients must not have had any radiation therapy or chemotherapy for medulloblastoma prior to study enrollment
- Patients must have a Lansky performance status of >/=30 for children </=10 years of age or a Karnofsky performance status of > 30 for children > 10 years of age.
- Participants must have normal organ and marrow function as defined below:
- Hemoglobin greater than 10 g/dL (can be transfused). Hemoglobin <10 g/dL due to operative blood loss is permitted.
- Absolute neutrophil count > 1.0x109/L
- Platelets > 100,000/uL (non-transfused)
- Total bilirubin <1.5 x upper limit normal
- SGOT (AST) or SGPT (ALT) <2.5 x upper limit normal (ULN) for age
- Creatinine clearance or radioisotope GFR >70 ml/min/1.73m2 or normal serum creatinine for patient's age and gender
- All females of child-bearing age must have a negative pregnancy test before being enrolled on study. All patients of child-bearing age must practice an effective method of birth control whilst undergoing chemotherapy on study.
- No history of allergic reactions attributed to compounds of similar chemical or biologic composition to cisplatin, lomustine, vincristine or cyclophosphamide.

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): NCT02212574
United States, Arizona | |
Phoenix Childrens Hospital Hematology/Oncology | |
Phoenix, Arizona, United States, 85016-7710 | |
United States, Colorado | |
Children's Hospital Colorado Center for Cancer & Blood Disorders | |
Aurora, Colorado, United States, 80045 | |
United States, Florida | |
M D Anderson Cancer Center-Orlando Pediatric Hematology/Oncology | |
Orlando, Florida, United States, 32806 | |
All Children's Hospital Pediatric Hematology/Oncology | |
Saint Petersburg, Florida, United States, 33701 | |
United States, Georgia | |
Children's Healthcare of Atlanta- Egleston Pediatric Neuro-Oncology | |
Atlanta, Georgia, United States, 30322 | |
United States, Illinois | |
Ann and Robert H Lurie Children's Hospital of Chicago Hematology/Oncology | |
Chicago, Illinois, United States, 60611 | |
United States, Maryland | |
Johns Hopkins University | |
Baltimore, Maryland, United States, 21287 | |
United States, Massachusetts | |
Dana-Farber Cancer Institute | |
Boston, Massachusetts, United States, 02215 | |
United States, Missouri | |
Washington University School of Medicine Pediatric Hematology/Oncology | |
Saint Louis, Missouri, United States, 63110 | |
United States, New Jersey | |
Hackensack University Medical Center | |
Hackensack, New Jersey, United States, 07601 | |
United States, New York | |
Memorial Sloan Kettering Cancer Center | |
New York, New York, United States, 10065 | |
United States, North Carolina | |
Duke University Medical Center | |
Durham, North Carolina, United States, 27705 | |
United States, Ohio | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 | |
United States, Oregon | |
Oregon Health and Science University Pediatric Hematology/Oncology | |
Portland, Oregon, United States, 97239-3098 | |
United States, Washington | |
Seattle Children's Hospital Hematology/Oncology | |
Seattle, Washington, United States, 98105 | |
United States, Wisconsin | |
Childrens Hospital of Wisconsin (Medical College of Wisconsin) | |
Milwaukee, Wisconsin, United States, 53226 |
Study Chair: | Kenneth Cohen, MD | Johns Hopkins University |
Responsible Party: | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
ClinicalTrials.gov Identifier: | NCT02212574 |
Other Study ID Numbers: |
J1403 NA_00091840 ( Other Identifier: JHMIRB ) |
First Posted: | August 8, 2014 Key Record Dates |
Last Update Posted: | January 12, 2021 |
Last Verified: | January 2021 |
Wnt Medulloblastoma Chemotherapy |
Medulloblastoma Glioma Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Neuroectodermal Tumors, Primitive Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Cyclophosphamide Lomustine Cisplatin Vincristine |
Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antirheumatic Agents Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Antineoplastic Agents, Phytogenic Tubulin Modulators Antimitotic Agents Mitosis Modulators |