Brain and Central Nervous System TumorsPhase 2NCT00003273
Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Brain Tumor
A Phase 2 study testing filgrastim, carboplatin and cisplatin in people with Brain and Central Nervous System Tumors and Neuroblastoma, run by NYU Langone Health at 19 sites in 3 countries.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Brain and Central Nervous System Tumors, Neuroblastoma, Retinoblastoma
- What is being tested?
- filgrastim, carboplatin and cisplatin
- Type of treatment
- Surgery or procedure
- How many people
- 0 participants
- Where
- 19 sitesin 3 countries
- Study dates
- Nov 1997to not given
- 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 Up to 10 years
- All sexes
- No healthy volunteers
- Child
DISEASE CHARACTERISTICS:
- Histologically confirmed malignant, newly diagnosed brain tumor
Regimen A:
- Posterior fossa medulloblastoma/primitive neuroectodermal tumor (PNET):
- All stages, under 3 years at diagnosis OR
- High stage (local residual tumor postoperatively and/or neuraxis or extraneural dissemination), 3-10 years at diagnosis
- Supratentorial PNET, pineoblastoma, cerebral neuroblastoma, ependymoblastoma, medulloepithelioma, medullomyoblastoma:
- All stages, under 10 years at diagnosis
- Brainstem PNET:
- All stages, irrespective of extent of resection, under 10 years at diagnosis
- Ependymoma or anaplastic ependymoma:
- All stages, any location (except primary spinal cord ependymoma), under 3 years at diagnosis OR
- Local residual tumor postoperatively and/or neuraxis dissemination, any location, 3-10 years at diagnosis
- Supratentorial ependymoma:
- All stages, irrespective of extent of resection, under 10 years at diagnosis, excluding gross totally resected (confirmed by postoperative MRI) low grade ependymoma not invading the ventricular system
- Metastatic retinoblastoma:
- Previously untreated (except for cryosurgery or laser surgery), under 10 years at presentation of metastatic disease
- Primary atypical teratoid/rhabdoid tumors of the CNS:
- Under 10 years at diagnosis
- Choroid plexus carcinoma:
- Incompletely resected, all sites, under 10 years at diagnosis
Regimen C:
- Anaplastic astrocytoma, glioblastoma multiforme, anaplastic oligodendroglioma, anaplastic ganglioglioma, other anaplastic mixed gliomas:
- Under 10 years at diagnosis
- Diffuse intrinsic pontine tumors:
- Unbiopsied, under 10 years at diagnosis
The following diagnoses are not eligible:
- Myxopapillary ependymoma of the spinal cord, low grade brainstem astrocytoma, primary CNS lymphoma or solid leukemic lesion (i.e., chloroma, granulocytic sarcoma), or primary CNS germ cell tumor
PATIENT CHARACTERISTICS:
Age:
- Under 10 at diagnosis
Performance status:
- Not specified
Life expectancy:
- Not specified
Hematopoietic:
- Not specified
Hepatic:
- Bilirubin less than 1.5 mg/dL
- SGPT less than 2.5 times upper limit of normal
Renal:
- Creatinine clearance greater than 60 mL/min
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- No prior chemotherapy
Endocrine therapy:
- Prior corticosteroids allowed
- No concurrent corticosteroids for the sole purpose of antiemesis
Radiotherapy:
- No prior radiotherapy
Surgery:
- See Disease Characteristics
- Recovered from prior surgery
Trial locations and contacts
19 sites in 3 countries.
19 sites in 3 countries
United States17 sites
Spectrum Health and DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Cancer Research Center of Hawaii
Honolulu, Hawaii, 96813
University of Wisconsin Comprehensive Cancer Center
Madison, Wisconsin, 53792-6164
Winthrop University Hospital
Mineola, New York, 11501
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, 10016
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021
Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Beth Israel Hospital North
New York, New York, 10128
Children's Hospital of Omaha
Omaha, Nebraska, 68114
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Maine Children's Cancer Program
Scarborough, Maine, 04074-9308
State University of New York Health Sciences Center - Stony Brook
Stony Brook, New York, 11790-7775
State University of New York - Upstate Medical University
Syracuse, New York, 13210
Albert Einstein Clinical Cancer Center
The Bronx, New York, 10461
St. Vincent Mercy Medical Center
Toledo, Ohio, 43608
Canada1 site
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
About this study
Official title: Dose Intensive Chemotherapy for Children Less Than Ten Years of Age Newly-Diagnosed With Malignant Brain Tumors: A Pilot Study of Two Alternative Intensive Induction Chemotherapy Regimens, Followed by Consolidation With Myeloablative Chemotherapy (Thiotepa, Etoposide and Carboplatin) and Autologous Stem Cell Rescue
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.
PURPOSE: Phase II trial to study the effectiveness of different regimens of combination chemotherapy followed by peripheral stem cell transplantation in treating children who have newly diagnosed brain tumor.
Read the detailed description
OBJECTIVES:
- Investigate the toxicity of and response rate to an intensification of an induction chemotherapy regimen (regimen A: cisplatin, vincristine, cyclophosphamide, and etoposide) by incorporation of high-dose methotrexate with leucovorin calcium rescue in patients with primitive neuroectodermal tumors and evidence of leptomeningeal dissemination (M1, M2, or M3).
- Investigate the toxicity of and response rate to a new dose intensive induction chemotherapy regimen (regimen C: vincristine, carboplatin, and temozolomide) in children under ten years of age who are newly diagnosed with either high grade gliomas or diffuse intrinsic pontine tumors. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Investigate the feasibility of utilizing regimen C chemotherapy followed by consolidation with myeloablative chemotherapy and autologous stem cell (either bone marrow and/or peripheral blood) rescue in these patients. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Investigate the toxicity of and response rate to an intensification of induction regimen A chemotherapy by incorporation of high-dose methotrexate with leucovorin calcium rescue in patients with primitive neuroectodermal tumors and evidence of leptomeningeal dissemination (M1, M2, or M3).
- Estimate the time to disease progression and the pattern of relapse in patients who do not have radiographic or cytologic evidence of residual disease at the time of consolidation chemotherapy and who, therefore, do not receive post consolidation irradiation.
- Estimate the time to disease progression and the pattern of relapse in patients who have radiographic or cytologic evidence of residual disease at the time of consolidation chemotherapy and who, therefore, receive post consolidation irradiation.
- Assess the morbidity and mortality of the consolidation chemotherapy regimen following either regimen C or the intensified regimen A in these patients. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Assess the impact that irradiation avoidance or the administration of reduced volume craniospinal and/or focused field local irradiation has on neuropsychometric, endocrinological functions, and physical growth.
OUTLINE: This is a two regimen study based on disease characteristics.
Patients in regimens A, B, and C undergo leukapheresis after receiving filgrastim (G-CSF) by subcutaneous (SC) injections.
- Regimen A: Patients without evidence of neuraxis dissemination receive five 21 day courses of the following chemotherapy: cisplatin IV over 6 hours on day 0; etoposide and cyclophosphamide IV over 1 hour on days 1 and 2; vincristine IV on days 0, 7, and 14 of courses 1, 2, and 3; and G-CSF SC beginning on day 3 of each course and continuing until blood counts recover or up to 48 hours before the start of the next course. Patients with evidence of neuraxis dissemination also receive high-dose methotrexate IV over 4 hours on day 3 and leucovorin calcium orally or by IV bolus starting 24 hours prior to methotrexate and continuing every 6 hours until methotrexate levels have diminished.
- Regimen B (closed to accrual effective 3/30/2000): Patients receive three 21-28 day courses of the following chemotherapy: vincristine IV on days 0, 7, and 14 of each course; carboplatin IV over 4 hours on days 3 and 4 of each course; oral procarbazine daily on days 0-4; oral lomustine on days 3 and 4; and G-CSF SC daily beginning 24 hours following the last dose of carboplatin and continuing until blood counts recover or up to 48 hours before the start of the next course. On day 7 of each course, patients also receive peripheral blood stem cell (PBSC) reinfusion following chemotherapy. Oral lomustine is administered only for the first two courses.
- Regimen C (open to accrual effective 07/21/2000): Patients receive four 28 day courses of the following chemotherapy: carboplatin IV over 4 hours on days 0 and 1 of each course; vincristine IV on days 0, 7, and 14 of the first three courses only; oral temozolomide daily on days 0-4; and G-CSF SC daily beginning on day 5 and continuing until blood counts recover.
After regimen A, B, or C and in the absence of disease progression, patients undergo consolidation myeloablative chemotherapy by receiving carboplatin IV over 4 hours on days -8, -7, and -6, and then thiotepa IV over 3 hours followed by etoposide IV on days -5, -4, and -3. Patients with malignant gliomas or unbiopsied diffuse intrinsic pontine tumors do not receive etoposide. On day 0, patients are reinfused with autologous PBSC. Following recovery from consolidation chemotherapy, patients with radiographic or cytologic evidence of residual disease undergo radiotherapy.
Patients are followed at 3 months, then every 3 months for the first 2 years, then every 6 months for years 2-4, and then annually thereafter.
PROJECTED ACCRUAL: Approximately 96 patients (73 for regimen A and 23 for regimen C) will be accrued for this study. (Regimen B closed to accrual effective 3/30/2000.)
- Being tested
- filgrastim, carboplatin, cisplatin, cyclophosphamide, etoposide
- Keywords
- childhood infratentorial ependymoma, childhood supratentorial ependymoma, localized resectable neuroblastoma, regional neuroblastoma, disseminated neuroblastoma, stage 4S neuroblastoma, extraocular retinoblastoma, childhood choroid plexus tumor, localized unresectable neuroblastoma, previously untreated childhood rhabdomyosarcoma, untreated childhood supratentorial primitive neuroectodermal tumor, untreated childhood medulloblastoma, newly diagnosed childhood ependymoma
- Medical subject headings
- Central Nervous System Neoplasms, Neuroblastoma, Retinoblastoma, Sarcoma, Choroid Plexus Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
carboplatinDrug
cisplatinDrug
cyclophosphamideDrug
etoposideDrug
methotrexateDrug
temozolomideDrug
thiotepaDrug
autologous bone marrow transplantationProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
radiation therapyRadiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startNov 1997
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 NYU Langone HealthThe 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
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jonathan L. Finlay, MB, ChB, Study chair, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003273
- Sponsor's protocol ID
- CDR0000066174
- Other IDs
- NYU-P9712, NCI-V98-1400
- Status last verified
- Nov 2015
- First submitted
- 1 Nov 1999
- Last updated
- 3 Dec 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose Intensive Chemotherapy for Children Less Than Ten Years of Age Newly-Diagnosed With Malignant Brain Tumors: A Pilot Study of Two Alternative Intensive Induction Chemotherapy Regimens, Followed by Consolidation With Myeloablative Chemotherapy (Thiotepa, Etoposide and Carboplatin) and Autologous Stem Cell Rescue
- ClinicalTrials.gov ID
- NCT00003273
- Acronym / sponsor ID
- CDR0000066174
- Keywords
- childhood infratentorial ependymoma, childhood supratentorial ependymoma, localized resectable neuroblastoma, regional neuroblastoma, disseminated neuroblastoma, stage 4S neuroblastoma, extraocular retinoblastoma, childhood choroid plexus tumor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- Nov 1997
- First posted
- 27 Jan 2003 (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: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.
PURPOSE: Phase II trial to study the effectiveness of different regimens of combination chemotherapy followed by peripheral stem cell transplantation in treating children who have newly diagnosed brain tumor.
Detailed description
OBJECTIVES:
- Investigate the toxicity of and response rate to an intensification of an induction chemotherapy regimen (regimen A: cisplatin, vincristine, cyclophosphamide, and etoposide) by incorporation of high-dose methotrexate with leucovorin calcium rescue in patients with primitive neuroectodermal tumors and evidence of leptomeningeal dissemination (M1, M2, or M3).
- Investigate the toxicity of and response rate to a new dose intensive induction chemotherapy regimen (regimen C: vincristine, carboplatin, and temozolomide) in children under ten years of age who are newly diagnosed with either high grade gliomas or diffuse intrinsic pontine tumors. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Investigate the feasibility of utilizing regimen C chemotherapy followed by consolidation with myeloablative chemotherapy and autologous stem cell (either bone marrow and/or peripheral blood) rescue in these patients. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Investigate the toxicity of and response rate to an intensification of induction regimen A chemotherapy by incorporation of high-dose methotrexate with leucovorin calcium rescue in patients with primitive neuroectodermal tumors and evidence of leptomeningeal dissemination (M1, M2, or M3).
- Estimate the time to disease progression and the pattern of relapse in patients who do not have radiographic or cytologic evidence of residual disease at the time of consolidation chemotherapy and who, therefore, do not receive post consolidation irradiation.
- Estimate the time to disease progression and the pattern of relapse in patients who have radiographic or cytologic evidence of residual disease at the time of consolidation chemotherapy and who, therefore, receive post consolidation irradiation.
- Assess the morbidity and mortality of the consolidation chemotherapy regimen following either regimen C or the intensified regimen A in these patients. (Regimen B closed to accrual effective 3/30/2000; regimen C open to accrual effective 7/21/2000)
- Assess the impact that irradiation avoidance or the administration of reduced volume craniospinal and/or focused field local irradiation has on neuropsychometric, endocrinological functions, and physical growth.
OUTLINE: This is a two regimen study based on disease characteristics.
Patients in regimens A, B, and C undergo leukapheresis after receiving filgrastim (G-CSF) by subcutaneous (SC) injections.
- Regimen A: Patients without evidence of neuraxis dissemination receive five 21 day courses of the following chemotherapy: cisplatin IV over 6 hours on day 0; etoposide and cyclophosphamide IV over 1 hour on days 1 and 2; vincristine IV on days 0, 7, and 14 of courses 1, 2, and 3; and G-CSF SC beginning on day 3 of each course and continuing until blood counts recover or up to 48 hours before the start of the next course. Patients with evidence of neuraxis dissemination also receive high-dose methotrexate IV over 4 hours on day 3 and leucovorin calcium orally or by IV bolus starting 24 hours prior to methotrexate and continuing every 6 hours until methotrexate levels have diminished.
- Regimen B (closed to accrual effective 3/30/2000): Patients receive three 21-28 day courses of the following chemotherapy: vincristine IV on days 0, 7, and 14 of each course; carboplatin IV over 4 hours on days 3 and 4 of each course; oral procarbazine daily on days 0-4; oral lomustine on days 3 and 4; and G-CSF SC daily beginning 24 hours following the last dose of carboplatin and continuing until blood counts recover or up to 48 hours before the start of the next course. On day 7 of each course, patients also receive peripheral blood stem cell (PBSC) reinfusion following chemotherapy. Oral lomustine is administered only for the first two courses.
- Regimen C (open to accrual effective 07/21/2000): Patients receive four 28 day courses of the following chemotherapy: carboplatin IV over 4 hours on days 0 and 1 of each course; vincristine IV on days 0, 7, and 14 of the first three courses only; oral temozolomide daily on days 0-4; and G-CSF SC daily beginning on day 5 and continuing until blood counts recover.
After regimen A, B, or C and in the absence of disease progression, patients undergo consolidation myeloablative chemotherapy by receiving carboplatin IV over 4 hours on days -8, -7, and -6, and then thiotepa IV over 3 hours followed by etoposide IV on days -5, -4, and -3. Patients with malignant gliomas or unbiopsied diffuse intrinsic pontine tumors do not receive etoposide. On day 0, patients are reinfused with autologous PBSC. Following recovery from consolidation chemotherapy, patients with radiographic or cytologic evidence of residual disease undergo radiotherapy.
Patients are followed at 3 months, then every 3 months for the first 2 years, then every 6 months for years 2-4, and then annually thereafter.
PROJECTED ACCRUAL: Approximately 96 patients (73 for regimen A and 23 for regimen C) will be accrued for this study. (Regimen B closed to accrual effective 3/30/2000.)
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| filgrastim | Biological | – |
| carboplatin | Drug | – |
| cisplatin | Drug | – |
| cyclophosphamide | Drug | – |
| etoposide | Drug | – |
| leucovorin calcium | Drug | – |
| methotrexate | Drug | – |
| temozolomide | Drug | – |
| thiotepa | Drug | – |
| vincristine sulfate | Drug | – |
| autologous bone marrow transplantation | Procedure or surgery | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
| radiation therapy | Radiation | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2015.
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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.
Jonathan L. Finlay, MB, ChB study chair
NYU Langone Health
19 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim, carboplatin and cisplatin in people with Brain and Central Nervous System Tumors and Neuroblastoma, run by NYU Langone Health at 19 sites in 3 countries.
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: withdrawn, as of the sponsor's last update on 3 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Argentina, Canada. 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 was withdrawn before enrolling participants, so there are no results.
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.
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 3 Dec 2015.