MedulloblastomaEarly Phase 1NCT02212574
Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma
A Early Phase 1 study testing Lomustine, Vincristine and Cisplatin in people with Medulloblastoma, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 16 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Medulloblastoma
- What is being tested?
- Lomustine, Vincristine and Cisplatin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 16 sitesUnited States
- Study dates
- 4 Apr 2017to 9 Nov 2018
- Phase
- Early Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 21Show fewer
- 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.
Who cannot join
None listed.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Children's Healthcare of Atlanta- Egleston Pediatric Neuro-Oncology
Atlanta, Georgia, 30322
Children's Hospital Colorado Center for Cancer & Blood Disorders
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21287
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Ann and Robert H Lurie Children's Hospital of Chicago Hematology/Oncology
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Duke University Medical Center
Durham, North Carolina, 27705
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Childrens Hospital of Wisconsin (Medical College of Wisconsin)
Milwaukee, Wisconsin, 53226
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
M D Anderson Cancer Center-Orlando Pediatric Hematology/Oncology
Orlando, Florida, 32806
Phoenix Childrens Hospital Hematology/Oncology
Phoenix, Arizona, 85016-7710
Oregon Health and Science University Pediatric Hematology/Oncology
Portland, Oregon, 97239-3098
Seattle Children's Hospital Hematology/Oncology
Seattle, Washington, 98105
Washington University School of Medicine Pediatric Hematology/Oncology
St Louis, Missouri, 63110
All Children's Hospital Pediatric Hematology/Oncology
St. Petersburg, Florida, 33701
About this study
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
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.
Read the 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.
- Conditions
- Medulloblastoma
- Being tested
- Lomustine, Vincristine, Cisplatin, Cyclophosphamide, Mesna
- Keywords
- Wnt, Medulloblastoma, Chemotherapy
- Medical subject headings
- Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LomustineDrug
Also called CCNU
Chemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1.
VincristineDrug
Also called Oncovin
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.
CisplatinDrug
Also called Cisplatinum
Chemotherapy Cycle A Cisplatin is given directly into a vein over 8 hours on Day 1.
CyclophosphamideDrug
Also called Cytoxan
Chemotherapy Cycle B Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2.
MesnaDrug
Also called Mesnex
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.
VincristineDrug
Also called Oncovin
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.
| Study group | Role | What they receive |
|---|---|---|
| Chemotherapy | Other | 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 Receives: Drug: Lomustine; Drug: Vincristine; Drug: Cisplatin; Drug: Cyclophosphamide; Drug: Mesna |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 years
Secondary outcomes (1)
Patterns of Failure
To evaluate the patterns of failure in those children that do not have progressive disease, progression free survival and overall survival.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2014 (estimated)
- Study start4 Apr 2017
- Primary completion9 Nov 2018
- Study completion9 Nov 2018
Why it stopped: Participant relapses led to abrupt stop of study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for MedulloblastomaStudies of the same condition, open and under way.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- The Matthew Larson Foundation for Pediatric Brain Tumors
- Responsible party
- Sponsor
- Investigators
- Kenneth Cohen, MD, Study chair, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02212574
- Sponsor's protocol ID
- J1403
- Other IDs
- NA_00091840
- Status last verified
- Oct 2021
- First submitted
- 7 Jul 2014
- Last updated
- 5 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02212574
- Acronym / sponsor ID
- J1403
- Conditions
- Medulloblastoma
- Keywords
- Wnt, Medulloblastoma, Chemotherapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Participant relapses led to abrupt stop of study.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- The Matthew Larson Foundation for Pediatric Brain Tumors
- Study start
- 4 Apr 2017 (actual)
- Primary completion
- 9 Nov 2018 (actual)
- Study completion
- 9 Nov 2018 (actual)
- First posted
- 8 Aug 2014 (estimated)
- Results
- Posted 5 Nov 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Chemotherapy | Other | Drug: Lomustine, Drug: Vincristine, Drug: Cisplatin, Drug: Cyclophosphamide, Drug: MesnaChemotherapy 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 |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LomustineAlso: CCNUChemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1. | Drug | Chemotherapy |
| VincristineAlso: OncovinChemotherapy 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. | Drug | Chemotherapy |
| CisplatinAlso: CisplatinumChemotherapy Cycle A Cisplatin is given directly into a vein over 8 hours on Day 1. | Drug | Chemotherapy |
| CyclophosphamideAlso: CytoxanChemotherapy Cycle B Cyclophosphamide is given into a vein over 1 hour on Days 1 and 2. | Drug | Chemotherapy |
| MesnaAlso: MesnexChemotherapy 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. | Drug | Chemotherapy |
| VincristineAlso: OncovinChemotherapy 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. | Drug | Chemotherapy |
Primary outcome
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; the number of participants who with progression free survival.
1 secondary outcome
Patterns of Failure
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth Cohen, MD study chair
Johns Hopkins University
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Lomustine, Vincristine and Cisplatin in people with Medulloblastoma, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 16 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression Free Survival (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2021.