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CRAD001CUS224T, Phase II Study of Everolimus (RAD001, AFINITOR®) for Children with Recurrent or Progressive Ependymoma

Study ID: STU 052014-038

Summary

This study is a Phase ii Study of everolimus (RaD001, afinitor[RegisteredTM]) for Children with Recurrent or Progressive ependymoma. eligible patients will have a diagnosis of a recurrent or progressive ependymoma for which there is no known effective therapy. All enrolled patients will be treated with everolimus at the previously established pediatric Phase i dose: 4.5 mg/m2/day. Patients will be monitored by MRi of the brain and spine for tumor response. The Primary aim of the study is to determine if everolimus has anti-tumor activity against pediatric ependymoma. Secondary aims include the duration of tumor response to everolimus, event-free survival, overall survival and treatment-related toxicities. in the first stage of the study, 7 patients will be accrued. if there is no response seen in these 7 patients, the study will be stopped. if a response is observed in the first 7 patients, an 11 additional patients will be accrued for a total of 18 patients.

Participant Eligibility

-- Diagnosis and Age: Ependymoma (WHO grade II) or Anaplastic Ependymoma (WHO grade III) that has relapsed or become refractory to standard therapy. Patients must have had histologic verification of their malignancy at original diagnosis or time of recurrence. Age must be >= 2 years and <= 21 years of age at study entry. -- Performance status: Lansky >= 50% for patients <= 10 years of age or Karnofsky >= 50% for patients > 10 years of age. -- Adequate bone marrow function as shown by: ANC >= 1,000/mm3, platelets >= 100,000/mm3 and hemoglobin > 9.0 g/dL. Note: lab parameters must be transfusion-independent -- Adequate liver function as shown by: -- Total serum bilirubin <= 2.0 mg/dL. -- ALT and AST <= 2.5x ULN -- INR <= 2. -- Adequate renal function: serum creatinine <= 1.5x ULN. -- Fasting serum cholesterol <= 300 mg/dL OR <= 7.75 mmol/L AND fasting triglycerides <= 2.5x ULN. NOTE: In case one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication. -- Signed informed consent obtained prior to any screening procedures. -- Patients must have measurable residual disease, defined as tumor that is measurable in two diameters on MRI. Diffuse leptomeningeal disease is not considered measurable. -- Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy prior to participating in this trial. -- No prior myelosupressive chemotherapy for at least 21 days prior to study enrollment. -- Must not have received craniospinal radiation therapy within 24 weeks prior to study entry and no involved field radiation therapy for 12 weeks prior to study enrollment. -- If patients received prior monoclonal antibody treatment, at least three half-lives must be elapsed by the time of treatment initiation. -- No investigational drugs for 4 weeks prior to study enrollment. -- No prior therapy with mTOR inhibitors (including sirolimus, temsirolimus or everolimus). -- Must be on stable doses or declining doses of corticosteroids for at least a week prior to study enrollment. -- MRI of the brain and the complete spine: All patients must have had an MRI of the brain and spine that has measurable tumor (not only diffuse leptomeningeal tumor) within14 days prior to study enrollment. Note: Central review of MRIs is required, but may be completed concurrently with patient registration. Completion of central review is not required prior to starting treatment --If available, tumor tissue, from either initial diagnosis or subsequent surgery, on Flash FrozenFormalin Fixed Paraffin Embedded (FFPE) slides (n = 12) to be submitted to the Pathology Laboratory at Children[Single Quote]s Medical Center-Dallas for correlative biological studies (including phosphorylated S6235/236, phosphorylated S6240/244, phosphorylated 4EBP1, phosphorylated PRAS40 (pT246), phosphorylated P70S6K, and PTEN expression.)

Cancer Related
Healthy Volunteers
No
UT Southwestern Principal Investigator
Daniel C Bowers

Contact:

Scott Baier

scott.baier@childrens.com

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