Study Stopped
Participant relapses led to abrupt stop of study.
Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma
Pilot Study Assessing the Feasibility of a Surgery and Chemotherapy-Only Approach in the Upfront Therapy of Children With Wnt Positive Standard Risk Medulloblastoma
2 other identifiers
interventional
6
1 country
16
Brief Summary
Participants enrolling on this study will receive standard of care chemotherapy for Wnt positive medulloblastoma without the radiation therapy or the weekly chemotherapy that is given during radiation therapy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for early_phase_1
Started Apr 2017
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 7, 2014
CompletedFirst Posted
Study publicly available on registry
August 8, 2014
CompletedStudy Start
First participant enrolled
April 4, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 9, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
November 9, 2018
CompletedResults Posted
Study results publicly available
November 5, 2021
CompletedNovember 5, 2021
October 1, 2021
1.6 years
July 7, 2014
October 8, 2021
October 8, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Progression Free Survival
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; the number of participants who with progression free survival.
3 years
Secondary Outcomes (1)
Patterns of Failure
3 years
Study Arms (1)
Chemotherapy
OTHERChemotherapy 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
Interventions
Chemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1.
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.
Chemotherapy Cycle A Cisplatin is given directly into a vein over 8 hours on Day 1.
Chemotherapy Cycle B Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2.
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.
Eligibility Criteria
You may qualify if:
- 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
- +6 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (16)
Phoenix Childrens Hospital Hematology/Oncology
Phoenix, Arizona, 85016-7710, United States
Children's Hospital Colorado Center for Cancer & Blood Disorders
Aurora, Colorado, 80045, United States
M D Anderson Cancer Center-Orlando Pediatric Hematology/Oncology
Orlando, Florida, 32806, United States
All Children's Hospital Pediatric Hematology/Oncology
St. Petersburg, Florida, 33701, United States
Children's Healthcare of Atlanta- Egleston Pediatric Neuro-Oncology
Atlanta, Georgia, 30322, United States
Ann and Robert H Lurie Children's Hospital of Chicago Hematology/Oncology
Chicago, Illinois, 60611, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Washington University School of Medicine Pediatric Hematology/Oncology
St Louis, Missouri, 63110, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Science University Pediatric Hematology/Oncology
Portland, Oregon, 97239-3098, United States
Seattle Children's Hospital Hematology/Oncology
Seattle, Washington, 98105, United States
Childrens Hospital of Wisconsin (Medical College of Wisconsin)
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Two of the participants relapsed not long into the study and the Data and Safety Management Committee (DSMC) recommended early closure of the study.
Results Point of Contact
- Title
- Dr. Kenneth Cohen
- Organization
- Johns Hopkins University
Study Officials
- STUDY CHAIR
Kenneth Cohen, MD
Johns Hopkins University
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 7, 2014
First Posted
August 8, 2014
Study Start
April 4, 2017
Primary Completion
November 9, 2018
Study Completion
November 9, 2018
Last Updated
November 5, 2021
Results First Posted
November 5, 2021
Record last verified: 2021-10