EpendymomaPhase 2NCT01088035
Carboplatin as a Radiosensitizer in Treating Childhood Ependymoma
A Phase 2 study testing Carboplatin in people with Ependymoma, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
This is a phase II study evaluating the feasibility of concurrent carboplatin given with focal radiation therapy in children age 12 months to < 21 years with newly diagnosed localized ependymoma who have no or minimal residual disease post-operatively (< 0.5 cm). Read more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- Ependymoma
- What is being tested?
- Carboplatin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2010to Apr 2015
- 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 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be enrolled before treatment begins.
- Patients must be ≥ 12 months and < 22 years of age at the time of diagnosis.
- The target tumors are primary brain non-metastatic (M0) ependymomas tumors. Patients must have histologic verification of an ependymoma at diagnosis. Patients with the following world health organization (WHO) diagnoses will be eligible for this study:Ependymoma (Subtypes: cellular, papillary, clear cell and tanycytic) and Anaplastic Ependymoma
- Life expectancy of ≥ 8 weeks.
- Newly diagnosed ependymoma and must not have had any prior chemotherapy or radiotherapy.
- All patients must have:
Show all 26Show fewer
- A pre-operative MRI scan of the brain with and without contrast. NOTE: CT scans are NOT sufficient for study eligibility since radiation therapy planning and responses will be based on MRI scans only.
- Post-operative head MRI scan with and without contrast (preferably within 72 hours post-operatively).
- Spinal MRI (T-1 weighted imaging with and without gadolinium) is required within 28 days of surgery if done post-operatively and within 14 days of surgery if done pre-operatively. For posterior fossa tumors, pre-operative MRI scans are preferred because surgically induced inflammation/blood can be difficult to distinguish from tumor.
- Lumbar CSF cytology examination obtained between 7 and 31 days following surgery.
- Adequate bone marrow function, defined as:
- Peripheral absolute neutrophil count (ANC) >1500/μL
- Platelet count ≥ 100,000/μL (transfusion independent)
- Hemoglobin ≥ 10.0 gm/dl (may receive RBC transfusions)
- Adequate renal function defined as:
- Serum creatinine < 1.5 times the upper limit of normal based on the patient's age, or creatinine clearance greater than 60 ml/min/1.73 m² corrected for age and body surface area.
- Adequate liver function defined as:
- Total bilirubin <1.5 x the institutional normal
- SGOT (AST) or SGPT (ALT) <2.5 x institutional normals.
- Patients must begin chemoradiotherapy within 56 days of definitive surgery.
- All patients and/or their parents or legal guardians must sign a written informed consent
- Patients must provide assent as per local IRB guidelines (if applicable).
- Patients and/or their families must consent to the mandatory biology studies, including serum Survivin levels, CSF Survivin levels, paraffin-embedded tissue and fresh-frozen tissue when available.
- Karnofsky/Lansky scoring greater than 50.
- Corticosteroid supportive care is permissible at the clinician's discretion prior to and during chemo-radiotherapy.
- Anti-seizure medication support is permitted as necessary and at the treating physician's discretion.
Who cannot join
- Prior chemotherapy or prior radiotherapy
- Patients who are pregnant or breast feeding, or patients (male or female) not employing adequate contraception. Acceptable means of birth control include IUD, oral contraceptive, subdermal implant, a condom with a contraceptive sponge or suppository or abstinence.
- Patients who are unable to undergo MR imaging
- Patients with evidence of metastatic disease on spine MRI or CSF sampling
- Patients with post-operative residual tumor > 0.5 cm, unless a repeat surgery is performed making the residual tumor less than 1.5 cm². Note: Timing for enrollment and initiation of therapy will begin after second surgery if a repeat surgery is performed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Memorial Hospital
Chicago, Illinois, 60614
About this study
Official title: A Phase II Multi-Institutional Trial of Focal Radiotherapy With Concomitant Carboplatin as a Radiosensitizer and the Prospective Analysis of Survivin, an Inhibitor of Apoptosis, as a Biomarker in Children With Newly Diagnosed Non-Metastatic Ependymoma and Minimal Residual Disease Post-Operatively
This is a phase II study evaluating the feasibility of concurrent carboplatin given with focal radiation therapy in children age 12 months to < 21 years with newly diagnosed localized ependymoma who have no or minimal residual disease post-operatively (< 0.5 cm). The hypothesis is that utilizing carboplatin as a radiosensitizer is feasible and tolerable and may improve event-free survival (EFS) and minimize local recurrences as compared to historic controls. Following a neurosurgical resection and staging, patients who meet the eligibility criteria will receive standard fractionated radiation therapy at doses of 54 to 59.4 Gy to the primary site depending upon age. All patients will receive 35 mg/m²/day of carboplatin prior to each fraction of radiotherapy. Although significant neutropenia is not anticipated, G-CSF will be administered per study guidelines during radiation if neutropenia occurs. All patients will be followed for toxicity, response (resolution of residual disease) and event-free survival (EFS). Patients' tumor sample, blood and cerebro-spinal fluid (CSF) will also be prospectively evaluated to quantify the level of Survivin, a known inhibitor of apoptosis, via immunohistochemistry, Western Blot Analysis (in tumor tissue) and ELISA (in blood and CSF). The feasibility of obtaining these levels prospectively and in real time will be evaluated.
- Conditions
- Ependymoma
- Being tested
- Carboplatin
- Keywords
- Childhood ependymoma, Ependymoma, Survivin
- Medical subject headings
- Ependymoma, Familial ependymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarboplatinDrug
Patients will receive daily carboplatin as a radiation sensitizer prior to radiation each day.
| Study group | Role | What they receive |
|---|---|---|
| Carboplatin | Experimental | Not described Receives: Drug: Carboplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure the EFS of newly diagnosed non-metastatic ependymoma patients treated with a combination of daily carboplatin as a radiosensitizer and conformal radiotherapy.
Measured: 3 years
Explore the tolerability, feasibility and toxicities associated with daily carboplatin as a radiosensitizer in patients with newly diagnosed non-metastatic ependymoma receiving focal fractionated radiotherapy.
Tolerability and toxicity will be measured by documenting the toxicities associated with this treatment using the current CTCAE version 4.0. The feasibility of this therapy will be measured by observing the ability to finish therapy without delays and the ability to receive daily carboplatin and radiation in the time alloted by the study (within 4 hours) on a daily basis. Delays in therapy will be documented in an effort to define both tolerability and feasibility.
Measured: 1 year
Secondary outcomes (6)
Explore the overall survival (OS) in patients with newly diagnosed non-metastatic ependymoma treated with this regimen.
Measured: 3 years
Evaluate the feasibility of quantifying Survivin expression in primary ependymoma tumor, blood and CSF samples in a prospective fashion.
Measured: 6 months
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and tumor histology.
Measured: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and patient responses.
Measured: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and survival outcomes.
Measured: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) tumor recurrences.
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 posted17 Mar 2010 (estimated)
- Study startApr 2010
- Primary completionJan 2015
- Study completionApr 2015 (estimated)
Why it stopped: Poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpendymomaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Brown University
- Responsible party
- Principal investigator, Jason Fangusaro, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Jason Fangusaro, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of ChicagoRachel Altura, MD, Principal investigator, Brown University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01088035
- Sponsor's protocol ID
- CMH 09C4
- Status last verified
- Feb 2015
- First submitted
- 16 Mar 2010
- Last updated
- 2 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Multi-Institutional Trial of Focal Radiotherapy With Concomitant Carboplatin as a Radiosensitizer and the Prospective Analysis of Survivin, an Inhibitor of Apoptosis, as a Biomarker in Children With Newly Diagnosed Non-Metastatic Ependymoma and Minimal Residual Disease Post-Operatively
- ClinicalTrials.gov ID
- NCT01088035
- Acronym / sponsor ID
- CMH 09C4
- Conditions
- Ependymoma
- Keywords
- Childhood ependymoma, Ependymoma, Survivin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor accrual
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- Brown University
- Study start
- Apr 2010
- Primary completion
- Jan 2015 (actual)
- Study completion
- Apr 2015 (estimated)
- First posted
- 17 Mar 2010 (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.
This is a phase II study evaluating the feasibility of concurrent carboplatin given with focal radiation therapy in children age 12 months to < 21 years with newly diagnosed localized ependymoma who have no or minimal residual disease post-operatively (< 0.5 cm). The hypothesis is that utilizing carboplatin as a radiosensitizer is feasible and tolerable and may improve event-free survival (EFS) and minimize local recurrences as compared to historic controls. Following a neurosurgical resection and staging, patients who meet the eligibility criteria will receive standard fractionated radiation therapy at doses of 54 to 59.4 Gy to the primary site depending upon age. All patients will receive 35 mg/m²/day of carboplatin prior to each fraction of radiotherapy. Although significant neutropenia is not anticipated, G-CSF will be administered per study guidelines during radiation if neutropenia occurs. All patients will be followed for toxicity, response (resolution of residual disease) and event-free survival (EFS). Patients' tumor sample, blood and cerebro-spinal fluid (CSF) will also be prospectively evaluated to quantify the level of Survivin, a known inhibitor of apoptosis, via immunohistochemistry, Western Blot Analysis (in tumor tissue) and ELISA (in blood and CSF). The feasibility of obtaining these levels prospectively and in real time will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Carboplatin | Experimental | Drug: Carboplatin |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (26 listed)
- Patients must be enrolled before treatment begins.
- Patients must be ≥ 12 months and < 22 years of age at the time of diagnosis.
- The target tumors are primary brain non-metastatic (M0) ependymomas tumors. Patients must have histologic verification of an ependymoma at diagnosis. Patients with the following world health organization (WHO) diagnoses will be eligible for this study:Ependymoma (Subtypes: cellular, papillary, clear cell and tanycytic) and Anaplastic Ependymoma
- Life expectancy of ≥ 8 weeks.
- Newly diagnosed ependymoma and must not have had any prior chemotherapy or radiotherapy.
Key exclusion criteria (5 listed)
- Prior chemotherapy or prior radiotherapy
- Patients who are pregnant or breast feeding, or patients (male or female) not employing adequate contraception. Acceptable means of birth control include IUD, oral contraceptive, subdermal implant, a condom with a contraceptive sponge or suppository or abstinence.
- Patients who are unable to undergo MR imaging
- Patients with evidence of metastatic disease on spine MRI or CSF sampling
- Patients with post-operative residual tumor > 0.5 cm, unless a repeat surgery is performed making the residual tumor less than 1.5 cm². Note: Timing for enrollment and initiation of therapy will begin after second surgery if a repeat surgery is performed.
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 |
|---|---|---|
| CarboplatinPatients will receive daily carboplatin as a radiation sensitizer prior to radiation each day. | Drug | Carboplatin |
Primary outcome
Measure the EFS of newly diagnosed non-metastatic ependymoma patients treated with a combination of daily carboplatin as a radiosensitizer and conformal radiotherapy.
Time frame: 3 years
Explore the tolerability, feasibility and toxicities associated with daily carboplatin as a radiosensitizer in patients with newly diagnosed non-metastatic ependymoma receiving focal fractionated radiotherapy.
Time frame: 1 year
Tolerability and toxicity will be measured by documenting the toxicities associated with this treatment using the current CTCAE version 4.0. The feasibility of this therapy will be measured by observing the ability to finish therapy without delays and the ability to receive daily carboplatin and radiation in the time alloted by the study (within 4 hours) on a daily basis. Delays in therapy will be documented in an effort to define both tolerability and feasibility.
6 secondary outcomes
Explore the overall survival (OS) in patients with newly diagnosed non-metastatic ependymoma treated with this regimen.
Time frame: 3 years
Evaluate the feasibility of quantifying Survivin expression in primary ependymoma tumor, blood and CSF samples in a prospective fashion.
Time frame: 6 months
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and tumor histology.
Time frame: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and patient responses.
Time frame: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) and survival outcomes.
Time frame: 3 years
Explore trends or relationships between Survivin expression (in tumor, blood and CSF samples) tumor recurrences.
Time frame: 3 years
Results and safety
The study was stopped early (reason given: Poor accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 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.
Jason Fangusaro, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
Rachel Altura, MD principal investigator
Brown University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Carboplatin in people with Ependymoma, run by Ann & Robert H Lurie Children's Hospital of Chicago 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 26 inclusion and 5 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 2 Mar 2015. 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 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 stopped early (reason given: Poor accrual). Results are not available in the source record.
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?
Measure the EFS of newly diagnosed non-metastatic ependymoma patients treated with a combination of daily carboplatin as a radiosensitizer and conformal radiotherapy. (time frame: 3 years). The study lists 2 primary outcomes in all. 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 2 Mar 2015.