Trial Search Results

Everolimus for Children With Recurrent or Progressive Ependymoma

The purpose of this study is to evaluate the anti-tumor activity of Everolimus among children with recurrent or progressive ependymoma. Recurrent or progressive ependymoma is incurable and has very limited treatment options. The rationale for this study is based upon both pre-clinical and clinical considerations: Immunohistochemistry studies have demonstrated that 20 out of 23 (87%) pediatric ependymomas are immunoreactive for phosphorylated S6, a biomarker that often predicts response to mTOR pathway-targeted therapy. Furthermore, children with with multiply recurrent ependymomas have had objective and durable responses to the mTOR inhibitor, Sirolimus (Rapamune, Pfizer). As a result of this pre-clinical and clinical data, this study will further investigate the activity of an mTOR pathway inhibitor, Everolimus, against children with recurrent or progressive ependymomas. In this study, Everolimus will be administered at a dose and schedule that have previously been demonstrated as safe and effective in children. Children may take Everolimus for up to 2 years on this study, until tumor progression or unacceptable toxicity.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

University of Texas Southwestern Medical Center

Stanford Investigator(s):

Intervention(s):

  • Drug: Everolimus

Phase:

Phase 2

Eligibility


Inclusion Criteria:

   1. 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.

   2. Tumor tissue must be available (from either time of initial diagnosis or relapse) and
   submitted for central pathology review and correlative biological studies.

   3. Performance status: Lansky ≥ 50% for patients ≤ 10 years of age or Karnofsky ≥ 50% for
   patients > 10 years of age.

   4. Adequate bone marrow, liver and renal function.

   5. Fasting serum cholesterol ≤ 300 mg/dL OR ≤ 7.75 mmol/L AND fasting triglycerides ≤ 2.5
   x the upper limit of normal. 6. Patients must have measurable residual disease,
   defined as tumor that is measurable in two diameters on MRI. Diffuse leptomeningeal
   disease is not considered measurable.

   6. 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 myelosuppressive chemotherapy for 28 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.

   7. MRI of the brain and the complete spine: All patients must have an MRI of the brain
   and spine that has measurable tumor (not only diffuse leptomeningeal tumor) within two
   weeks prior to study enrollment.

Exclusion Criteria:

   1. Prior treatment with Everolimus or other rapamycin analogs (e.g. sirolimus,
   temsirolimus).

   2. Concommitant use of medications known to have inhibition or induction of CYP3A
   enzymes. Systemic corticosteroids (e.g., dexamethasone is a CYP3A inducer) are not
   allowed. Inhaled corticosteroids are allowed.

   3. Known impairment of gastrointestinal (GI) function or GI disease that may
   significantly alter the absorption of oral Everolimus.

   4. Uncontrolled diabetes mellitus as defined by HbA1c > 8% despite adequate therapy.
   Patients with a known history of impaired fasting glucose or diabetes mellitus may be
   included, however blood glucose and antidiabetic treatment must be monitored closely
   throughout the trial and adjusted as necessary.

Ages Eligible for Study

2 Years - 21 Years

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
CCTO
650-498-7061
Not Recruiting