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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 : Recruiting
First Posted : August 8, 2014
Last Update Posted : October 6, 2017
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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.

Condition or disease Intervention/treatment Phase
Medulloblastoma Drug: Lomustine Drug: Vincristine Drug: Cisplatin Drug: Cyclophosphamide Drug: Mesna Not Applicable

Detailed Description:

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
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
Study Start Date : March 2015
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2019

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

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


Contacts
Contact: Kenneth Cohen Cohen, MD 410-614-5055 kcohen@jhmi.edu
Contact: Tammy Scott, RN 410-614-5990 scottta@jhmi.edu

Locations
United States, Arizona
Phoenix Childrens Hospital Hematology/Oncology Recruiting
Phoenix, Arizona, United States, 85016-7710
Contact: Michael Etzl, Dr    602-546-0920    metzl@phoenixchildrens.com   
United States, Colorado
Children's Hospital Colorado Center for Cancer & Blood Disorders Recruiting
Aurora, Colorado, United States, 80045
Contact: Kathleen Dorris, M.D., M.S.    720-777-6772    kathleen.dorris@childrenscolorado.org   
United States, Florida
M D Anderson Cancer Center-Orlando Pediatric Hematology/Oncology Not yet recruiting
Orlando, Florida, United States, 32806
Contact: Amy Smith, Dr    321-841-8588    amy.smith@orlandohealth.com   
All Children's Hospital Pediatric Hematology/Oncology Recruiting
Saint Petersburg, Florida, United States, 33701
Contact: Stacie Stapleton, Dr    727-767-4176    Stacie.Stapleton@allkids.org   
United States, Georgia
Children's Healthcare of Atlanta- Egleston Pediatric Neuro-Oncology Recruiting
Atlanta, Georgia, United States, 30322
Contact: Tobey MacDonald, Dr    404-727-1447    tobey.macdonald@emory.edu   
United States, Illinois
Ann and Robert H Lurie Children's Hospital of Chicago Hematology/Oncology Recruiting
Chicago, Illinois, United States, 60611
Contact: Stewart Goldman, Dr    312-227-4844    sgoldman@northwestern.edu   
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21287
Contact: Kenneth Cohen, MD, MBA    410-614-5055    kcohen@jhmi.edu   
Contact: Tammy Scott, RN    4140-614-5990    scottta@jhmi.edu   
United States, Massachusetts
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Pratiti Bandopadhayay, MBBS, PhD         
United States, Missouri
Washington University School of Medicine Pediatric Hematology/Oncology Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Joshua Rubin, Dr    314-454-6018    rubin_j@kids.wustl.edu   
United States, New Jersey
Hackensack University Medical Center Recruiting
Hackensack, New Jersey, United States, 07601
Contact: Derek Hanson, MD    551-996-5437    dhanson@hackensackumc.org   
United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Kevin De Braganca, MD    212-639-3449    debragak@mskcc.org   
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Mohamed S AbdelBaki, MBBCh    614-722-3250    mohamed.abdelbaki@nationwidechildrens.org   
United States, Oregon
Oregon Health and Science University Pediatric Hematology/Oncology Not yet recruiting
Portland, Oregon, United States, 97239-3098
Contact: Kellie Nazemi, Dr    503-494-0714    nazemik@ohsu.edu   
United States, Washington
Seattle Children's Hospital Hematology/Oncology Recruiting
Seattle, Washington, United States, 98105
Contact: Sarah Leary, Dr    206-987-2106    sarah.leary@seattlechildrens.org   
United States, Wisconsin
Childrens Hospital of Wisconsin (Medical College of Wisconsin) Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Jeffrey Knipstein, MD    414-955-4108    jknipstein@jhmi.edu   
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Investigators
Study Chair: Kenneth Cohen, MD Johns Hopkins University

Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT02212574     History of Changes
Other Study ID Numbers: J1403
NA_00091840 ( Other Identifier: JHMIRB )
First Posted: August 8, 2014    Key Record Dates
Last Update Posted: October 6, 2017
Last Verified: October 2017

Keywords provided by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins:
Wnt
Medulloblastoma
Chemotherapy

Additional relevant MeSH terms:
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
Cisplatin
Cyclophosphamide
Vincristine
Lomustine
Mesna
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Antineoplastic Agents, Phytogenic
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators
Protective Agents