Brain and Central Nervous System TumorsPhase 2NCT00003846
Radiation Therapy, Chemotherapy, and Peripheral Stem Cell Transplantation in Treating Patients With Primitive Neuroectodermal Tumors
A Phase 2 study testing filgrastim, carboplatin and cyclophosphamide in people with Brain and Central Nervous System Tumors and Neuroblastoma, run by Children's Oncology Group at 13 sites in United States.
Trial summary
RATIONALE: Radiation therapy uses x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of radiation therapy and chemotherapy and kill more tumor cells. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Brain and Central Nervous System Tumors, Neuroblastoma
- What is being tested?
- filgrastim, carboplatin and cyclophosphamide
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 13 sitesUnited States
- Study dates
- Jul 1999to Mar 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Histologically proven primitive neuroectodermal tumor (PNET) of one of the following types:
- Atypical teratoid/rhabdoid tumor
- Medulloblastoma
- Desmoplastic medulloblastoma
- Ependymoblastoma
- Medullomyoblastoma
- Spongioblastoma
- Spongioblastoma polare
- Primitive polar spongioblastoma
- Medulloepithelioma
- Neuroblastoma
- Pineoblastoma
- Posterior fossa PNET must be M1-3 or M0 with greater than 1.5 cm2 residual disease
- Non posterior fossa PNET and other types must be M0-3
- If M3, must show clear evidence of tumor on MRI
- No marrow involvement or other extraneural metastases
- No M4 disease
- No cord compression requiring emergency radiotherapy
PATIENT CHARACTERISTICS:
Age:
- 3 to 21
Performance status:
- Not specified
Life expectancy:
- Not specified
Hematopoietic:
- Absolute neutrophil count at least 1,000/mm^3
- Platelet count at least 150,000/mm^3 (no platelet transfusions)
- Hemoglobin at least 10 g/dL (red blood cell transfusions allowed)
Hepatic:
- Bilirubin less than 1.5 times upper limit of normal (ULN)
- AST or ALT less than 2.5 times ULN
Renal:
- Creatinine clearance or glomerular filtration rate at least 70 mL/min
Cardiovascular:
- Shortening fraction greater than 27% by echocardiogram OR
- Ejection fraction greater than 47% by MUGA
Pulmonary:
- FEV_1/FVC greater than 60% except for children who:
- Are uncooperative
- Have no dyspnea at rest
- Have no exercise intolerance
- Have pulse oximetry greater than 94% on room air
Other:
- Not pregnant or nursing
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- Not specified
Endocrine therapy:
- Steroids for increased intracranial pressure allowed
Radiotherapy:
- See Disease Characteristics
- No prior urgent radiotherapy
Surgery:
- Not specified
Other:
- No prior therapy for tumor
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Children's Hospital Medical Center - Cincinnati
Cincinnati, Ohio, 45229-3039
Children's Hospital of Columbus
Columbus, Ohio, 43205-2696
Children's Hospital of Denver
Denver, Colorado, 80218
University of Texas - MD Anderson Cancer Center
Houston, Texas, 77030
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700
Jonsson Comprehensive Cancer Center, UCLA
Los Angeles, California, 90095-1781
University of Minnesota Cancer Center
Minneapolis, Minnesota, 55455
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, 10016
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021
Children's Hospital of Orange County
Orange, California, 92668
Oregon Cancer Center at Oregon Health Sciences University
Portland, Oregon, 97201-3098
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Children's National Medical Center
Washington D.C., District of Columbia, 20010-2970
About this study
Official title: Treatment of High Risk Central Nervous System Embryonal Tumors With Conventional Radiotherapy and Intensive Consolidation Chemotherapy With Peripheral Blood Progenitor Cell (PBSC) Support
RATIONALE: Radiation therapy uses x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of radiation therapy and chemotherapy and kill more tumor cells.
PURPOSE: Phase II trial to study the effectiveness of radiation therapy, chemotherapy and peripheral stem cell transplantation in treating patients with primitive neuroectodermal tumors.
Read the detailed description
OBJECTIVES:
- Determine the safety of postradiotherapy high-dose consolidation chemotherapy with peripheral blood stem cell (PBSC) support in patients with high-risk primitive neuroectodermal tumors.
- Determine the safety of delaying radiotherapy by approximately one month in these patients.
- Determine the maximum tolerated dose of thiotepa in these patients.
- Determine the toxic effects of intensive chemotherapy with PBSC support in these patients.
- Assess the time to hematopoietic recovery after PBSC infusion when intensive chemotherapy is used after craniospinal radiotherapy in these patients.
- Determine the overall and event-free survival of patients treated with this regimen.
OUTLINE: This is a dose-escalation study of thiotepa during consolidation therapy.
- Induction: Within 31 days of initial surgery, patients receive induction therapy comprising vincristine IV on day 0, cyclophosphamide IV over 2 hours on days 0 and 1, and filgrastim (G-CSF) subcutaneously (SC) beginning on day 2 and continuing for at least 7-10 days. Peripheral blood stem cells (PBSC) are then collected.
- Chemoradiotherapy: After blood cell counts recover, and within 28 days of starting induction, patients begin chemoradiotherapy. Patients receive vincristine IV once weekly for 8 doses. Radiotherapy is administered 5 days a week, for 6 weeks, beginning within the same week as the start of vincristine.
- Consolidation: Therapy begins 4-6 weeks after the last radiation treatment in the absence of disease progression. The first and third course are the same and comprise vincristine IV on day 0, carboplatin IV over 1 hour on days 0 and 1, thiotepa IV over 3 hours on days 2-4, and G-CSF SC daily beginning on day 7. PBSC are reinfused on day 7. The second course comprises vincristine IV on day 0, carboplatin IV over 1 hour on days 0 and 1, cyclophosphamide IV over 2 hours on days 2 and 3, and G-CSF SC daily beginning on day 5. PBSC are reinfused on day 5. Each course lasts 21 days.
For consolidation therapy, cohorts of 6-12 patients each receive escalating doses of thiotepa until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which no more than 2 of 12 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 2 years, and then annually thereafter.
PROJECTED ACCRUAL: A total of 24-56 patients will be accrued for this study.
- Being tested
- filgrastim, carboplatin, cyclophosphamide, thiotepa, vincristine sulfate
- Keywords
- regional neuroblastoma, disseminated neuroblastoma, stage 4S neuroblastoma, untreated childhood supratentorial primitive neuroectodermal tumor, untreated childhood medulloblastoma, newly diagnosed childhood ependymoma
- Medical subject headings
- Central Nervous System Neoplasms, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Also called Neupogen®, G-CSF, NSC#614629
carboplatinDrug
Also called Paraplatin®, CBDCA, NSC#241240
cyclophosphamideDrug
Also called Cytoxan®, Neosar®, Procytox®, CPM
thiotepaDrug
Also called TRIETHYLENETHIOPHOSPHORAMIDE, THIOPLEX®, TEPA, NSC# 6396
Also called Oncovin®, Vincasar®, leucocristine, VCR
bone marrow ablation with stem cell supportProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
radiation therapyRadiation
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | See detailed description. Receives: Biological: filgrastim; Drug: carboplatin; Drug: cyclophosphamide; Drug: thiotepa; Drug: vincristine sulfate; Procedure: bone marrow ablation with stem cell support; Procedure: peripheral blood stem cell transplantation; Radiation: radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event Free Survival
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2004 (estimated)
- Study startJul 1999
- Primary completionOct 2004
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain and Central Nervous System TumorsStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- H. Stacy Nicholson, MD, MPH, Study chair, OHSU Knight Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003846
- Sponsor's protocol ID
- 99702
- Other IDs
- COG-99702, CCG-99702, CDR0000067006
- Status last verified
- Jul 2014
- First submitted
- 1 Nov 1999
- Last updated
- 28 Jul 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of High Risk Central Nervous System Embryonal Tumors With Conventional Radiotherapy and Intensive Consolidation Chemotherapy With Peripheral Blood Progenitor Cell (PBSC) Support
- ClinicalTrials.gov ID
- NCT00003846
- Acronym / sponsor ID
- 99702
- Keywords
- regional neuroblastoma, disseminated neuroblastoma, stage 4S neuroblastoma, untreated childhood supratentorial primitive neuroectodermal tumor, untreated childhood medulloblastoma, newly diagnosed childhood ependymoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jul 1999
- Primary completion
- Oct 2004 (actual)
- Study completion
- Mar 2007 (actual)
- First posted
- 25 May 2004 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Radiation therapy uses x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of radiation therapy and chemotherapy and kill more tumor cells.
PURPOSE: Phase II trial to study the effectiveness of radiation therapy, chemotherapy and peripheral stem cell transplantation in treating patients with primitive neuroectodermal tumors.
Detailed description
OBJECTIVES:
- Determine the safety of postradiotherapy high-dose consolidation chemotherapy with peripheral blood stem cell (PBSC) support in patients with high-risk primitive neuroectodermal tumors.
- Determine the safety of delaying radiotherapy by approximately one month in these patients.
- Determine the maximum tolerated dose of thiotepa in these patients.
- Determine the toxic effects of intensive chemotherapy with PBSC support in these patients.
- Assess the time to hematopoietic recovery after PBSC infusion when intensive chemotherapy is used after craniospinal radiotherapy in these patients.
- Determine the overall and event-free survival of patients treated with this regimen.
OUTLINE: This is a dose-escalation study of thiotepa during consolidation therapy.
- Induction: Within 31 days of initial surgery, patients receive induction therapy comprising vincristine IV on day 0, cyclophosphamide IV over 2 hours on days 0 and 1, and filgrastim (G-CSF) subcutaneously (SC) beginning on day 2 and continuing for at least 7-10 days. Peripheral blood stem cells (PBSC) are then collected.
- Chemoradiotherapy: After blood cell counts recover, and within 28 days of starting induction, patients begin chemoradiotherapy. Patients receive vincristine IV once weekly for 8 doses. Radiotherapy is administered 5 days a week, for 6 weeks, beginning within the same week as the start of vincristine.
- Consolidation: Therapy begins 4-6 weeks after the last radiation treatment in the absence of disease progression. The first and third course are the same and comprise vincristine IV on day 0, carboplatin IV over 1 hour on days 0 and 1, thiotepa IV over 3 hours on days 2-4, and G-CSF SC daily beginning on day 7. PBSC are reinfused on day 7. The second course comprises vincristine IV on day 0, carboplatin IV over 1 hour on days 0 and 1, cyclophosphamide IV over 2 hours on days 2 and 3, and G-CSF SC daily beginning on day 5. PBSC are reinfused on day 5. Each course lasts 21 days.
For consolidation therapy, cohorts of 6-12 patients each receive escalating doses of thiotepa until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which no more than 2 of 12 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 2 years, and then annually thereafter.
PROJECTED ACCRUAL: A total of 24-56 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Biological: filgrastim, Drug: carboplatin, Drug: cyclophosphamide, Drug: thiotepa, Drug: vincristine sulfate, Procedure: bone marrow ablation with stem cell support, Procedure: peripheral blood stem cell transplantation, Radiation: radiation therapySee detailed description. |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| filgrastimAlso: Neupogen®, G-CSF, NSC#614629 | Biological | Treatment |
| carboplatinAlso: Paraplatin®, CBDCA, NSC#241240 | Drug | Treatment |
| cyclophosphamideAlso: Cytoxan®, Neosar®, Procytox®, CPM | Drug | Treatment |
| thiotepaAlso: TRIETHYLENETHIOPHOSPHORAMIDE, THIOPLEX®, TEPA, NSC# 6396 | Drug | Treatment |
| vincristine sulfateAlso: Oncovin®, Vincasar®, leucocristine, VCR | Drug | Treatment |
| bone marrow ablation with stem cell support | Procedure or surgery | Treatment |
| peripheral blood stem cell transplantation | Procedure or surgery | Treatment |
| radiation therapy | Radiation | Treatment |
Primary outcome
Event Free Survival
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2014.
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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.
H. Stacy Nicholson, MD, MPH study chair
OHSU Knight Cancer Institute
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim, carboplatin and cyclophosphamide in people with Brain and Central Nervous System Tumors and Neuroblastoma, run by Children's Oncology Group at 13 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?
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: finished, as of the sponsor's last update on 28 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Event Free Survival. 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 28 Jul 2014.