Childhood Choroid Plexus TumorPhase 3NCT01987596
Study of Fixed vs. Flexible Filgrastim to Accelerate Bone Marrow Recovery After Chemotherapy in Children With Cancer
A Phase 3 study testing filgrastim in people with Childhood Choroid Plexus Tumor and Childhood Medulloblastoma, run by Barbara Ann Karmanos Cancer Institute at 1 site in United States.
Trial summary
This randomized phase III trial studies flexible administration of filgrastim after combination chemotherapy to see how well it works compared to fixed administration of filgrastim in decreasing side effects of chemotherapy in younger patients with cancer. Read more about this study
- Who is it for?
- Ages 1 year to 25 years
- Conditions the study is for
- Childhood Choroid Plexus Tumor, Childhood Medulloblastoma, Childhood Pineoblastoma
- What is being tested?
- filgrastim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Jun 2018
- Phase
- Phase 3
- 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 1 year to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects must have or have had at initial diagnosis, histologic proof of their malignancy; young children with primary embryonal brain tumor treated according to Head Start protocol are eligible; subjects with bone marrow involvement are NOT eligible for study
- Patients will receive repeated cycles of identical chemotherapy that will likely result in grades III-IV hematological toxicity; patients will be treated outside of Children's Oncology Group (COG) protocols with specific requirements for schedule of G-CSF administration; the following categories of patients treated at Children's Hospital of Michigan are eligible for this study:
- Patients with brain tumors treated according to Head Start II protocol with vincristine, etoposide, cyclophosphamide, and cisplatin (OPEC) chemotherapy;
- Patients with recurrent Hodgkin lymphoma treated with ICE (ifosfamide, carboplatin, etoposide) chemotherapy;
- Patients with recurrent solid tumors including sarcomas, Wilms' tumor, neuroblastomas, or brain tumors treated with high dose ICE or ICT (ifosfamide, carboplatin, topotecan) chemotherapy
- Patients with UH Wilms' tumor treated with CE (cyclophosphamide, etoposide); patients with neuroblastoma treated with CE (carboplatin, etoposide);
Show all 23Show fewer
- Patients with soft tissue sarcomas treated with IA (ifosfamide, doxorubicin);
- Patients with osteosarcoma treated with high dose ifosfamide
- Subjects must have fully recovered from the toxic effects of any prior therapy; at least 3 weeks should have elapsed since the last dose of chemotherapy (6 weeks in the case of nitrosourea containing therapy); subjects must have recovered from previous colony-stimulating factor therapy and have been off colony-stimulating factors (G-CSF, granulocyte macrophage colony-stimulating factor [GM-CSF], interleukin [IL]-11) for more than 10 days and off erythropoietin for 30 days
- ANC > 1000/uL
- Platelet count > 100,000/uL
- Creatinine clearance or glomerular filtration rate (GFR) which is greater than or equal to 70 ml/min/1.73 m^2
- Bilirubin less than 1.5 x normal limit (NL)
- Serum glutamic oxaloacetic transaminase (SGOT) or serum glutamate pyruvate transaminase (SGPT) less than 2.5 x NL for age
- Subjects should have a normal ejection fraction (per institutional limits), no evidence of cardiac arrhythmias requiring therapy, and a fractional shortening of > 28%
- All subjects must have a life expectancy of 12 weeks or more
- Diagnostic categories
- Sarcoma (soft tissue and bone)
- Kidney tumors
- Brain tumors
- Other solid tumors (gonadal and germ cell tumors, retinoblastoma, neuroblastoma, and miscellaneous tumors)
- Hodgkin lymphoma
- Performance status must be > 60 from Lansky (age 1 to 16) or Karnofsky (age > 16)
Who cannot join
- Subjects with any of the following will NOT be eligible for study:
- Bone marrow involvement
- Active myelogenous leukemia, or history of myelogenous leukemia
- Pregnancy
Trial locations and contacts
1 site in 1 country.
United States1 site
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201
About this study
Official title: Prospective and Randomized Study of Fixed Versus Flexible Prophylactic Administration of Granulocyte Colony-Stimulating Factor (G-CSF) in Children With Cancer
This randomized phase III trial studies flexible administration of filgrastim after combination chemotherapy to see how well it works compared to fixed administration of filgrastim in decreasing side effects of chemotherapy in younger patients with cancer. Cancer chemotherapy frequently results in neutropenia (low blood counts) when patients are susceptible to severe infections. A medicine called G-CSF (filgrastim) stimulates bone marrow and daily filgrastim shots are commonly used to shorten neutropenic periods and decrease infections after chemotherapy. Since filgrastim is customarily used on a fixed schedule starting early after chemotherapy and there are data that early doses may not be needed, this study tests new flexible schedule of filgrastim to optimize its use by reducing the number of painful shots, cost of treatment, and filgrastim side effects in children with cancer receiving chemotherapy.
Read the detailed description
PRIMARY OBJECTIVES:
- To compare the effect of flexible vs. fixed administration of G-CSF (filgrastim) on the parameters of hematological recovery including duration of absolute neutrophil count (ANC) < 500/uL; time to ANC recovery >= 1,000/uL and time to platelet recovery >= 75,000/uL in children receiving myelotoxic chemotherapy.
SECONDARY OBJECTIVES:
- To compare the effect of flexible vs. fixed administration of G-CSF on the incidence of febrile neutropenia and number of hospital days on antibiotics following myelotoxic chemotherapy.
II. To evaluate the number of days of platelet transfusion events after chemotherapy cycles with flexible vs. fixed administration of G-CSF.
III. To evaluate on the incidence and duration of G-CSF-related side effects including extremities/back pain and headaches after chemotherapy courses followed by flexible vs. fixed administration of G-CSF.
IV. To evaluate the peripheral blood progenitor responses and subsets of progenitor cells (cluster of differentiation [CD]34/41/61/117/10/19/11b/33) to chemotherapy followed by flexible vs. fixed administration of G-CSF.
OUTLINE:
CHEMOTHERAPY: Depending on their diagnosis patients are assigned to 1 of 3 chemotherapy regimens.
ICE: Patients receive etoposide intravenously (IV) over 1 hour on days 1-3, ifosfamide IV over 3 hours on days 1-3, and carboplatin IV over 1 hour on day 4. Patients with recurrent Hodgkin lymphoma receive etoposide and ifosfamide on days 1-3 and carboplatin on day 3.
ICT: Patients receive topotecan hydrochloride IV over 30 minutes on days 1-3, and ifosfamide and carboplatin as in ICE.
OPEC: Patients receive vincristine sulfate on days 1, 8, and 15; etoposide IV over 1 hour on days 1-3; cyclophosphamide IV over 1 hour on days 1-2; and cisplatin IV over 6 hours on day 4.
For all chemotherapy regimens, treatment repeats every 21 days for 2 courses. Patients are then randomized to 1 of 2 treatment arms.
ARM I (fixed filgrastim): Patients receive filgrastim subcutaneously (SC) once daily (QD) started at 24 hours after completion of chemotherapy and stopped when ANC reaches at least 1,000/uL post nadir.
ARM II (flexible filgrastim): Patients receive filgrastim SC QD started on the first day after chemotherapy when ANC falls below 1,000/uL and stopped when ANC reaches at least 1,000/uL post nadir.
After completion of the first filgrastim treatment, patients cross-over to the other filgrastim arm and repeat the same course of chemotherapy as before. After completion of the second filgrastim treatment, chemotherapy treatment may continue for up to 5 (OPEC) or 6 (ICE, ICT) courses in the absence of disease progression or unacceptable toxicity.
- Conditions
- Childhood Choroid Plexus Tumor, Childhood Medulloblastoma, Childhood Pineoblastoma, Childhood Soft Tissue Sarcoma, Childhood Supratentorial Primitive Neuroectodermal Tumor, Neuroblastoma
- Being tested
- filgrastim
- Medical subject headings
- Choroid Plexus Neoplasms, Medulloblastoma, Neuroblastoma, Osteosarcoma, Retinoblastoma, Wilms Tumor, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Also called G-CSF, Neupogen
Given SC once daily starting on day 1 after chemotherapy in Arm I (fixed) and on any day when ANC drops below 1000/mcl in Arm II (flexible)
| Study group | Role | What they receive |
|---|---|---|
| Arm I (fixed filgrastim) | Experimental | Patients receive filgrastim SC QD started at 24 hours after completion of chemotherapy and stopped when ANC reaches at least 1,000/uL post nadir. Receives: Biological: filgrastim |
| Arm II (flexible filgrastim) | Experimental | Patients receive filgrastim SC QD started on the first day after chemotherapy when ANC falls below 1,000/uL and stopped when ANC reaches at least 1,000/uL post nadir. Receives: Biological: filgrastim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Days to ANC Greater Than or Equal to 1,000/uL From the Start of Chemotherapy
Time in days until absolute neutrophil count (ANC) recovery to greater than or equal to 1,000/uL from the start of chemotherapy
Measured: From the start of the course until the first date the ANC reaches >= 1,000/uL post nadir, assessed up to 1 year
Secondary outcomes (3)
Incidence of Febrile Neutropenia
occurrence of febrile neutropenia
Measured: Up to 1 year
Cumulative GCSF Dose
Cumulative GCSF dose - number of GCSF injections until ANC recovery greater than or equal to 1,000/uL from the start of chemotherapy
Measured: up until engraftment
Days to First G-CSF Dose
Time (in days) to first G-CSF dose
Measured: time to ANC 1000
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2013 (estimated)
- Study startAug 2013
- Primary completionJun 2018
- Study completionJun 2018
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 Childhood Choroid Plexus TumorStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Barbara Ann Karmanos Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Children's Hospital of Michigan
- Responsible party
- Principal investigator, Maxim Yankelevich, Barbara Ann Karmanos Cancer Institute
- Investigators
- Maxim Yankelevich, Principal investigator, Barbara Ann Karmanos Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01987596
- Sponsor's protocol ID
- 2013-062
- Other IDs
- NCI-2013-02001, 2013-062, P30CA022453
- Status last verified
- Oct 2020
- First submitted
- 12 Nov 2013
- Last updated
- 29 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective and Randomized Study of Fixed Versus Flexible Prophylactic Administration of Granulocyte Colony-Stimulating Factor (G-CSF) in Children With Cancer
- ClinicalTrials.gov ID
- NCT01987596
- Acronym / sponsor ID
- 2013-062
- Conditions
- Childhood Choroid Plexus Tumor, Childhood Medulloblastoma, Childhood Pineoblastoma, Childhood Soft Tissue Sarcoma, Childhood Supratentorial Primitive Neuroectodermal Tumor, Neuroblastoma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Barbara Ann Karmanos Cancer InstituteUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Children's Hospital of Michigan
- Study start
- Aug 2013
- Primary completion
- Jun 2018 (actual)
- Study completion
- Jun 2018 (actual)
- First posted
- 19 Nov 2013 (estimated)
- Results
- Posted 29 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This randomized phase III trial studies flexible administration of filgrastim after combination chemotherapy to see how well it works compared to fixed administration of filgrastim in decreasing side effects of chemotherapy in younger patients with cancer. Cancer chemotherapy frequently results in neutropenia (low blood counts) when patients are susceptible to severe infections. A medicine called G-CSF (filgrastim) stimulates bone marrow and daily filgrastim shots are commonly used to shorten neutropenic periods and decrease infections after chemotherapy. Since filgrastim is customarily used on a fixed schedule starting early after chemotherapy and there are data that early doses may not be needed, this study tests new flexible schedule of filgrastim to optimize its use by reducing the number of painful shots, cost of treatment, and filgrastim side effects in children with cancer receiving chemotherapy.
Detailed description
PRIMARY OBJECTIVES:
- To compare the effect of flexible vs. fixed administration of G-CSF (filgrastim) on the parameters of hematological recovery including duration of absolute neutrophil count (ANC) < 500/uL; time to ANC recovery >= 1,000/uL and time to platelet recovery >= 75,000/uL in children receiving myelotoxic chemotherapy.
SECONDARY OBJECTIVES:
- To compare the effect of flexible vs. fixed administration of G-CSF on the incidence of febrile neutropenia and number of hospital days on antibiotics following myelotoxic chemotherapy.
II. To evaluate the number of days of platelet transfusion events after chemotherapy cycles with flexible vs. fixed administration of G-CSF.
III. To evaluate on the incidence and duration of G-CSF-related side effects including extremities/back pain and headaches after chemotherapy courses followed by flexible vs. fixed administration of G-CSF.
IV. To evaluate the peripheral blood progenitor responses and subsets of progenitor cells (cluster of differentiation [CD]34/41/61/117/10/19/11b/33) to chemotherapy followed by flexible vs. fixed administration of G-CSF.
OUTLINE:
CHEMOTHERAPY: Depending on their diagnosis patients are assigned to 1 of 3 chemotherapy regimens.
ICE: Patients receive etoposide intravenously (IV) over 1 hour on days 1-3, ifosfamide IV over 3 hours on days 1-3, and carboplatin IV over 1 hour on day 4. Patients with recurrent Hodgkin lymphoma receive etoposide and ifosfamide on days 1-3 and carboplatin on day 3.
ICT: Patients receive topotecan hydrochloride IV over 30 minutes on days 1-3, and ifosfamide and carboplatin as in ICE.
OPEC: Patients receive vincristine sulfate on days 1, 8, and 15; etoposide IV over 1 hour on days 1-3; cyclophosphamide IV over 1 hour on days 1-2; and cisplatin IV over 6 hours on day 4.
For all chemotherapy regimens, treatment repeats every 21 days for 2 courses. Patients are then randomized to 1 of 2 treatment arms.
ARM I (fixed filgrastim): Patients receive filgrastim subcutaneously (SC) once daily (QD) started at 24 hours after completion of chemotherapy and stopped when ANC reaches at least 1,000/uL post nadir.
ARM II (flexible filgrastim): Patients receive filgrastim SC QD started on the first day after chemotherapy when ANC falls below 1,000/uL and stopped when ANC reaches at least 1,000/uL post nadir.
After completion of the first filgrastim treatment, patients cross-over to the other filgrastim arm and repeat the same course of chemotherapy as before. After completion of the second filgrastim treatment, chemotherapy treatment may continue for up to 5 (OPEC) or 6 (ICE, ICT) courses in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (fixed filgrastim) | Experimental | Biological: filgrastimPatients receive filgrastim SC QD started at 24 hours after completion of chemotherapy and stopped when ANC reaches at least 1,000/uL post nadir. |
| Arm II (flexible filgrastim) | Experimental | Biological: filgrastimPatients receive filgrastim SC QD started on the first day after chemotherapy when ANC falls below 1,000/uL and stopped when ANC reaches at least 1,000/uL post nadir. |
Who may qualify
- Ages
- 1 year to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (23 listed)
- Subjects must have or have had at initial diagnosis, histologic proof of their malignancy; young children with primary embryonal brain tumor treated according to Head Start protocol are eligible; subjects with bone marrow involvement are NOT eligible for study
- Patients will receive repeated cycles of identical chemotherapy that will likely result in grades III-IV hematological toxicity; patients will be treated outside of Children's Oncology Group (COG) protocols with specific requirements for schedule of G-CSF administration; the following categories of patients treated at Children's Hospital of Michigan are eligible for this study:
- Subjects must have fully recovered from the toxic effects of any prior therapy; at least 3 weeks should have elapsed since the last dose of chemotherapy (6 weeks in the case of nitrosourea containing therapy); subjects must have recovered from previous colony-stimulating factor therapy and have been off colony-stimulating factors (G-CSF, granulocyte macrophage colony-stimulating factor [GM-CSF], interleukin [IL]-11) for more than 10 days and off erythropoietin for 30 days
- ANC > 1000/uL
- Platelet count > 100,000/uL
Key exclusion criteria (4 listed)
- Subjects with any of the following will NOT be eligible for study:
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: G-CSF, NeupogenGiven SC once daily starting on day 1 after chemotherapy in Arm I (fixed) and on any day when ANC drops below 1000/mcl in Arm II (flexible) | Biological | Arm I (fixed filgrastim), Arm II (flexible filgrastim) |
Primary outcome
Days to ANC Greater Than or Equal to 1,000/uL From the Start of Chemotherapy
Time frame: From the start of the course until the first date the ANC reaches >= 1,000/uL post nadir, assessed up to 1 year
Time in days until absolute neutrophil count (ANC) recovery to greater than or equal to 1,000/uL from the start of chemotherapy
3 secondary outcomes
Incidence of Febrile Neutropenia
Time frame: Up to 1 year
Cumulative GCSF Dose
Time frame: up until engraftment
Days to First G-CSF Dose
Time frame: time to ANC 1000
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2020.
- 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.
Maxim Yankelevich principal investigator
Barbara Ann Karmanos Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing filgrastim in people with Childhood Choroid Plexus Tumor and Childhood Medulloblastoma, run by Barbara Ann Karmanos Cancer Institute at 1 site 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 23 inclusion and 4 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 29 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 29 Oct 2020). 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?
Days to ANC Greater Than or Equal to 1,000/uL From the Start of Chemotherapy (time frame: From the start of the course until the first date the ANC reaches >= 1,000/uL post nadir, assessed up to 1 year). 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 29 Oct 2020.