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EpendymomaPhase 2NCT01295944

Carboplatin and Bevacizumab for Recurrent Ependymoma

A Phase 2 study testing Carboplatin and Bevacizumab in people with Ependymoma and Anaplastic Ependymoma, run by National Cancer Institute (NCI) at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The goal of this clinical research study is to learn if the combination of bevacizumab and carboplatin can help to control recurrent ependymoma. The safety of this drug combination will also be studied. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Ependymoma, Anaplastic Ependymoma
What is being tested?
Carboplatin and Bevacizumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
2 sitesUnited States
Study dates
27 Apr 2011to 14 May 2021
Phase
Phase 2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically proven intra-cranial or spinal ependymoma or anaplastic ependymoma. There must be pathologic or imaging confirmation of tumor progression or regrowth.
  • The patient must have at least 1 block of tissue or 15 unstained slides at a minimum available for central pathology review and molecular profiling of the tissue sample.
  • All patients must sign an informed consent indicating that they are aware of the investigational nature of this study. Patients must have signed an authorization for the release of their protected health information.
  • Patients must be greater than or equal to 18 years old.
  • Patients must have a Karnofsky performance status of > 60.
  • Patients must have adequate bone marrow function (white blood cell (WBC) greater than or equal to 3,000/microliter, absolute neutrophil count (ANC) greater than or equal to 1,500/mm^3, platelet count of greater than to equal to 100,000/mm^3, and hemoglobin greater than or equal to 10 gm/dl), adequate liver function (Serum glutamic oxaloacetic transaminase (SGOT) [aspartate aminotransferase (AST) <92.5 Units/L] and bilirubin less than or equal to 1.5 mg/dL), and adequate renal function (creatinine < 1.5 mg/dL and calculated creatinine clearance greater than or equal to 60 cc/min) before starting therapy. Eligibility level for hemoglobin may be reached by transfusion.
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  • Patients must have shown unequivocal radiographic evidence for tumor progression by magnetic resonance imaging (MRI) or computed tomography (CT) scan.
  • At the time of registration: Patients must have recovered from the toxic effects of prior therapy: greater than or equal to 28 days from any investigational agent, greater than or equal to 28 days from prior cytotoxic therapy, greater than or equal to 14 days from vincristine, greater than or equal to 42 days from nitrosoureas, greater than or equal to 21 days from procarbazine administration, and greater than or equal to 7 days for non-cytotoxic agents, e.g., interferon, tamoxifen, thalidomide, cis-retinoic acid, etc. (radiosensitizer does not count). Any questions related to the definition of non-cytotoxic agents should be directed to the Principal Investigator.
  • Patients having undergone recent resection of recurrent or progressive tumor will be eligible as long as all of the following conditions apply:
  • They have recovered from the effects of surgery.
  • A minimum of 28 days have elapsed from the day of surgery to the day of registration Step 2.
  • For core or needle biopsy, a minimum of 7 days must have elapsed prior to registration Step 2.
  • Residual disease following resection of recurrent ependymoma is not mandated for eligibility into the study. To best assess the extent of residual disease post-operatively, a CT/ MRI should be done no later than 96 hours in the immediate post-operative period or at least 4 weeks post-operatively, within 14 days prior to consent. If the within 96-hour after surgery scan is more than 14 days before consent the scan needs to be repeated. If the steroid dose is increased between the date of imaging and consent, a new baseline MRI/CT is required on a stable steroid dosage for at least 5 days.
  • Patients must have failed prior radiation therapy* and must have an interval of greater than or equal to 42 days from the completion of radiation therapy to study entry. Note: Patients with an indication for craniospinal radiotherapy (i.e., extensive leptomeningeal disease) but have refused palliative craniospinal radiotherapy are eligible.
  • Patients with prior therapy that included interstitial brachytherapy or stereotactic radiosurgery must have confirmation of true progressive disease rather than radiation necrosis based upon either positron emission tomography (PET) or Thallium scanning, MR spectroscopy, or surgical/pathological documentation of disease.
  • Women of childbearing potential must have a negative B (Beta)-human chorionic gonadotropin (HCG) pregnancy test documented within 14 days prior to registration.
  • Women of childbearing potential and male participants agree to practice adequate contraception.

Who cannot join

  • Patients with any significant medical illnesses that in the investigators opinion cannot be adequately controlled with appropriate therapy or would compromise the patients ability to tolerate this therapy.
  • Patients with a history of any other cancer (except non-melanoma skin cancer or carcinoma insitu of the cervix), unless in complete remission and off of all therapy for that disease for a minimum of 3 years are ineligible.
  • Patients with an active infection or serious intercurrent medical illness.
  • Patients found to be pregnant/breast feeding. Patients must not be pregnant because animal studies show that carboplatin and bevacizumab are teratogenic
  • Patients with any disease that will obscure toxicity or dangerously alter drug metabolism.
  • Patients who have received prior therapy with bevacizumab, or related drugs (previous therapy with carboplatin is allowed).
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  • Inadequately controlled hypertension (defined as systolic blood pressure >150 millimeters of mercury (mmHg) and/or diastolic blood pressure > 100 mmHg) despite antihypertensive medication.
  • New York Heart Association (NYHA) Grade II or greater congestive heart failure.
  • History of myocardial infarction or unstable angina within 12 months prior to Day 1.
  • History of stroke or transient ischemic attack.
  • Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent peripheral arterial thrombosis) within 6 months prior to Day 1.
  • History of hemoptysis (greater than or equal to 1/2 teaspoon of bright red blood per episode) within 1 month prior to Day 1.
  • Evidence of bleeding diathesis or significant coagulopathy (in the absence of therapeutic anticoagulation). (To be eligible, Prothrombin time/international normalized ratio (prothrombin time (PT) international normalized ratio (INR)) should be < 1.4 for patients not on warfarin.)
  • Patients receiving full-dose anticoagulants therapy (e.g., warfarin or Low-molecular-weigh (LMW) heparin) and does not meet both of the following criteria:
  • No active bleeding or pathological condition that carries a high risk of bleeding (e.g., tumor involving major vessels or known varices).
  • In-range INR (usually between 2 and 3) on a stable dose of oral anticoagulant or on a stable dose of low molecular weight heparin.
  • Major surgical procedure, open biopsy, or significant traumatic injury within 28 days prior to Day 1 of treatment or anticipation of need for major surgical procedure during the course of the study.
  • Core biopsy or other minor surgical procedure, excluding placement of a vascular access device, within 7 days prior to Day 1.
  • History of abdominal fistula or gastrointestinal perforation within 6 months prior to Day 1.
  • Serious, non-healing wound, active ulcer, or untreated bone fracture.
  • Proteinuria as demonstrated by a urine protein:creatinine (UPC) ratio greater than or equal to 1.0 at screening, or Urine dipstick for proteinuria greater than or equal to 2+ (patients discovered to have greater than or equal to 2+ proteinuria on dipstick urinalysis at baseline should undergo a 24 hour urine collection and must demonstrate less than or equal to 1g of protein in 24 hours to be eligible).
  • Known hypersensitivity to any component of bevacizumab.
  • Patients has current active hepatic or biliary disease (with exception of patients with Gilbert's syndrome, asymptomatic gallstones, or stable chronic liver disease per investigator assessment).

Trial locations and contacts

2 sites in 1 country.

United States2 sites
  • Massachusetts General Hospital/Dana Farber

    Boston, Massachusetts, 02114

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10021

Other trials in United States

About this study

Official title: Phase II Trial of Carboplatin and Bevacizumab for the Treatment of Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial and Spinal Cord Ependymoma in Adults: A Multi-Center Trial

The goal of this clinical research study is to learn if the combination of bevacizumab and carboplatin can help to control recurrent ependymoma. The safety of this drug combination will also be studied.

Read the detailed description

Background:

  • Ependymomas are glial based tumors arising from the ependymal lining of the ventricular system and central canal of the spinal cord
  • These tumors affect both adults and children and represent approximately 1.2%-7.8% of all intracranial cancers.
  • Currently, the standard therapy for newly diagnosed low-grade ependymoma includes total surgical excision, when possible, followed by radiation therapy. Complete surgical resection is often not possible because of the location of the tumor and the concern for damage to surrounding eloquent brain during surgery. The situation is even more critical for patients with anaplastic ependymomas because of the higher proliferative rate and greater propensity for tumor infiltration into surrounding normal brain, preventing any possibility of complete tumor removal by surgery.
  • For patients with the more aggressive anaplastic ependymoma, chemotherapy is often administered either before or after the radiation in the hope that infiltrating tumor cells will be eliminated.
  • Extensive experience has been gathered with the use of bevacizumab in other neuroepithelial tumors such as malignant gliomas. Based on the interesting results observed in the reported small series of patients with recurrent ependymomas treated with bevacizumab, as well as on the evidence of vascular endothelial growth factor (VEGF)-promoted angiogenesis in these tumors, we designed a phase II study to test the efficacy of bevacizumab in patients with recurrent ependymoma. As results in most types of tumors have indicated that anti-angiogenesis therapies are more effective when given in combination with cytotoxic chemotherapy, in this study bevacizumab will be combined with carboplatin. The choice of carboplatin is justified by the fact that, as detailed above, this remains the most effective agent in this disease, and extensive toxicity data is available for the combination of bevacizumab and carboplatin in a variety of tumor types, including glioblastomas (GBMs).

Objective:

To evaluate the efficacy of carboplatin and bevacizumab for the treatment of recurrent low grade or anaplastic ependymoma. The primary endpoint will be progression-free survival (PFS) at one year.

Design:

  • This is a phase II study to evaluate the efficacy of carboplatin and bevacizumab for the treatment of recurrent low grade or anaplastic ependymoma. This trial is designed utilizing a Simon optimal two-stage design.
  • Carboplatin will be given on day 1 of each cycle. Bevacizumab will be administered on days 1 and 15 of each cycle. The total duration of treatment will be 6 cycles. After cycle 6, carboplatin should be discontinued, but bevacizumab may be continued at the discretion of the treating physician.
  • Patients will be monitored for hematologic or serologic evidence of myelosuppression, hepatic injury, renal injury, and electrolyte disturbances and for clinical evidence of other toxicity.
Keywords
Chemotherapy, Brain Tumor, Spinal Cord Tumor, Malignancy, Neurologic
Medical subject headings
Ependymoma, Brain Neoplasms, Spinal Cord Neoplasms, Neoplasms, Neurologic Manifestations

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CarboplatinDrug

    Also called Paraplatin

    Carboplatin will be given on day 1 of each cycle; the carboplatin dose should be calculated using the Calvert formula: Carboplatin dose (mg) = target Area Under the Curve (AUC) x (Creatinine clearance (CrCl) + 25; The total duration of treatment will be 6 cycles. After cycle 6, carboplatin should be discontinued.

  • BevacizumabDrug

    Also called Avastin

    Bevacizumab will be administered on days 1 and 15 of each cycle. Bevacizumab will be administered at a dose of 10 mg/kg; The total duration of treatment will be 6 cycles. After cycle 6, bevacizumab may be continued at the discretion of the treating physician.

Study groups in NCT01295944
Study groupRoleWhat they receive
1/Carboplatin and Bevacizumab for Recurrent EpendymomaExperimentalThe total duration of treatment will be 6 cycles. After cycle 6, carboplatin should be discontinued, but bevacizumab may be continued at the discretion of the treating physician.
Receives: Drug: Carboplatin; Drug: Bevacizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants That Have Progressive Free Survival (PFS) After 1 Year

    Percentage of participants that have progressive disease after 1 year. Kaplan-Meier method is used for the analyses of PFS. Progression is a 25% increase in the sum of products of all measurable lesions over smallest sum observed (over baseline if no decrease) using the same techniques as baseline. Clear worsening of any evaluable disease, or appearance of any new lesion/site, clear clinical worsening or failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer).

    Measured: 1 year

Secondary outcomes (6)
  1. Number of Participants With a (Complete Response (CR) + Partial Response (PR))

    Response was measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete Response (CR) is complete disappearance of all measurable and evaluable disease. No new lesions. Partial Response (PR) is greater than or equal to 50% decrease under baseline in the sum of products of perpendicular diameters of all measurable lesions.

    Measured: Up to 6 months and 1 week

  2. Overall Survival (OS)

    OS was calculated by the Kaplan Meier methodology. OS is defined as the time from treatment start date until date of death or date last known alive.

    Measured: The time from treatment start date until date of death or date last known alive, up to 68 months

  3. Mean Core Symptom Severity With Daily Activities Using the MD Anderson Symptom Inventory for Brain Tumors (MDASI-BT)

    The MDASI-BT consists of 23 symptoms rated on an 11 point scale (0 - not present, to 10 - as bad as you can imagine) to indicate the presence and severity of the symptom. All participants with at least one valid questionnaire are included in the analyses. We calculated the mean core symptom severity score rated on the scale from 0-not present to 10-as bad as you can imagine at the time of clinical evaluation. Differences of at least 2 points will be classified as the minimum clinically meaningful change in the symptom severity measures. For example, an increase of 2 points or more would mean a moderate improvement, whereas a decrease of 2 points or more would be interpreted as moderate worsening.

    Measured: Completed at baseline, before a brain or spine magnetic resonance imaging (MRI)/clinical evaluation, and at cycle 2, cycle 4, and cycle 6 (each cycle is 28 days)

  4. Mean Core Symptom Severity With Daily Activities Using the MD Anderson Symptom Inventory for Spine Tumors (MDASI-SP) Instrument

    The MDASI-SP consists of 23 symptoms rated on an 11 point scale (0 - not present, to 10 - as bad as you can imagine) to indicate the presence and severity of the symptom. All participants with at least one valid questionnaire are included in the analyses. We calculated the mean core symptom severity score rated on the scale from 0-not present to 10-as bad as you can imagine at the time of clinical evaluation. Differences of at least 2 points will be classified as the minimum clinically meaningful change in the symptom severity measures. For example, an increase of 2 points or more would mean a moderate improvement, whereas a decrease of 2 points or more would be interpreted as moderate worsening.

    Measured: Completed at baseline, before a brain or spine magnetic resonance imaging (MRI)/clinical evaluation, and at cycle 2, cycle 4, and cycle 6 (each cycle is 28 days)

  5. Mean Symptom Interference With Daily Activities Using the MD Anderson Symptom Inventory for Brain Tumors (MDASI-BT)

    The MDASI-BT consists of 23 symptoms rated on an 11 point scale (0 - did not interfere, to 10 - interfered completely) to indicate the symptoms that interfere with a participants life in the last 24 hours. All participants with at least one valid questionnaire are included in the analyses. We calculated the mean core symptom interference score rated on the scale from 0- did not interfere, to 10- interfered completely at the time of clinical evaluation. Differences of at least 2 points will be classified as the minimum clinically meaningful change in the symptom interference measures. For example, an increase of 2 points or more would mean a moderate improvement, whereas a decrease of 2 points or more would be interpreted as moderate worsening.

    Measured: Completed at baseline, before a brain or spine magnetic resonance imaging (MRI)/clinical evaluation, and at cycle 2, cycle 4, and cycle 6 (each cycle is 28 days)

  6. Mean Symptom Interference With Daily Activities Using the MD Anderson Symptom Inventory for Spine Tumors (MDASI-SP) Instrument

    The MDASI-SP consists of 23 symptoms rated on an 11 point scale (0 - did not interfere, to 10 - interfered completely) to indicate the symptoms that interfere with a participants life in the last 24 hours. All participants with at least one valid questionnaire are included in the analyses. We calculated the mean core symptom interference score rated on the scale from 0- did not interfere, to 10- interfered completely at the time of clinical evaluation. Differences of at least 2 points will be classified as the minimum clinically meaningful change in the symptom interference measures. For example, an increase of 2 points or more would mean a moderate improvement, whereas a decrease of 2 points or more would be interpreted as moderate worsening.

    Measured: Completed at baseline, before a brain or spine magnetic resonance imaging (MRI)/clinical evaluation, and at cycle 2, cycle 4, and cycle 6 (each cycle is 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Feb 2011 (estimated)
  2. Study start27 Apr 2011
  3. Primary completion23 Sep 2020
  4. Study completion14 May 2021

Resources

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
35 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01295944
Sponsor's protocol ID
160009
Other IDs
16-C-0009
Status last verified
Jul 2025
First submitted
11 Feb 2011
Last updated
5 Aug 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: We will share coded, linked data in an National Institutes of Health-funded or approved public repository; coded, linked data in another public repository; coded, linked data in Biomedical Translational Research Information System (BTRIS), and identified or coded, linked data with approved outside collaborators under appropriate agreements.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Carboplatin and Bevacizumab in people with Ependymoma and Anaplastic Ependymoma, run by National Cancer Institute (NCI) at 2 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 17 inclusion and 23 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: finished, as of the sponsor's last update on 5 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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?

Yes. Results are posted in the registry (first posted 28 Dec 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?

Percentage of Participants That Have Progressive Free Survival (PFS) After 1 Year (time frame: 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 5 Aug 2025.

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