Know Cancer

or
forgot password

A Phase II Study of Everolimus (RAD001) for Children With Chemotherapy and/or Radiation-Refractory Progressive or Recurrent Low-Grade Gliomas


Phase 2
3 Years
21 Years
Not Enrolling
Both
Glioma, Low-grade Glioma

Thank you

Trial Information

A Phase II Study of Everolimus (RAD001) for Children With Chemotherapy and/or Radiation-Refractory Progressive or Recurrent Low-Grade Gliomas


- Participants will be given a study medication-dosing calendar for each treatment
course. Each treatment course lasts 4 weeks. Participants can remain on study for 12
courses (48 weeks). Participants will take RAD001 tablets orally once a day. If
participants cannot swallow tablets, participants will be instructed on how to create a
liquid preparation.

- During all treatment courses participants will have a physical exam. Each treatment
course lasts 4 weeks. A medical history, urine test, blood tests, ECG and tumor imaging
using MRI and MR Perfusion and Diffusion will also be performed. Blood sampling for
RAD001 levels will be drawn weekly during the first course (4 weeks) of treatment and
then drawn once during subsequent courses thereafter.

- Pharmacokinetic studies will be done to see how the body absorbs, distributes and
excretes RAD001 by measuring the amount of the drug in the body at different time
points after taking the medication. Blood will be drawn one time before the start of
RAD001 therapy. Blood samples will also be drawn on Day 1 of cycle 2 at the following
times: before the Day 1 dose (pre-dose), 2, and 5 hours after the Day 1 dose of RAD001.


Inclusion Criteria:



- Patients must have histologic verification of one of the eligible diagnoses listed
here: Astrocytoma variants; fibrillary, protoplasmic, mixed: Pilocytic astrocytoma;
including pilomyxoid variants: Pleomorphic xanthoastrocytoma: infantile desmoplastic
astrocytoma: ganglioglioma: oligodendroglial tumors: mixed glioma.

- Patients must have received at least one cancer-directed therapy and patients with
allergies to carboplatin must have demonstrated progressive disease after cessation
of therapy.

- Must have at least one measurable site of disease that has not been previously
irradiated. If the patient has previous irradiation to the marker lesion(s), there
must be evidence of progression since radiation treatment.

- Patients must be between 3 years of age and 21 years of age

- Karnofsky Performance Status of 50% or greater for patients less than 10 years of age
or Lansky Score of 50% or greater for patients 10 and up.

- Participants must have recovered from the acute toxic effects of all prior
chemotherapy or radiotherapy prior to entering the study. Refer to protocol for
specific time restrictions with prior therapy completion.

- Adequate bone marrow function as defined in protocol

- Adequate renal function as defined in protocol

- Adequate liver function as defined in protocol

- Patients must have a fasting LDL cholesterol within the normal range per
institutional guidelines

- Patients taking cholesterol lowering agent must be on a single medication and on a
stable dose for at least 4 weeks

- Fasting serum cholesterol as outlined in protocol

- Patients must not be taking enzyme-inducing anticonvulsants

- Patients may not be currently receiving strong inhibitors of CYP3A4

Exclusion Criteria:

- Presence of NF1 by clinical examination or by genetic testing

- Patients who have had a major surgery or significant traumatic injury within 2 weeks
of start of study drug, patients who have not recovered from teh side effects of any
major surgery, or patients that may require major surgery during the course of the
study

- Patients receiving chronic, systemic treatment with corticosteroids or another
immunosuppressive agent. Topical or inhaled are allowed

- Evidence of plexiform neurofibroma, malignant peripheral nerve sheath tumor, or other
cancer requiring treatment with chemotherapy or radiation therapy

- Uncontrolled brain or leptomeningeal metastases from plexiform neurofibromas,
malignant peripheral nerve sheath tumors, or other cancers (other than astrocytoma
variants; fibrillary, protoplasmic, mixed: Pilocytic astrocytoma; including
pilomyxoid variants: Pleomorphic xanthoastrocytoma: infantile desmoplastic
astrocytoma: ganglioglioma: oligodendroglial tumors: mixed glioma), including
patients who continue to require glucocorticoids for control of symptoms related to
brain or leptomeningeal metastases.

- Other malignancies within the past three years except for adequately treated
carcinoma of the cervix or basal or squamous cell carcinomas of the skin

- Patients who have any severe and/or uncontrolled medical conditions or other
conditions that could affect their participation in the study (see protocol for
examples)

- Known history of HIV seropositivity

- Impairment of gastrointestinal function or gastrointestinal disease that may
significantly alter the absorption of RAD001

- Active, bleeding diathesis or oral anti-vitamin K medication (except low dose
coumarin)

- Female patients who are pregnant or breast feeding

- Prior treatment with an mTOR inhibitor

- Known hypersensitivity to RAD001 or other rapamycins or to is excipients

- Dental braces or prosthesis that interferes with tumor imaging

- Patients with a positive history of Hepatitis B or Hepatitis C

- Patients should not receive immunization with attenuated live vaccines within one
week of study entry or during study period.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the response of children with chemotherapy-refractory or progressive low-grade gliomas to RAD001.

Outcome Time Frame:

3 years

Safety Issue:

No

Principal Investigator

Mark W. Kieran, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute

Authority:

United States: Food and Drug Administration

Study ID:

09-001

NCT ID:

NCT00782626

Start Date:

June 2009

Completion Date:

Related Keywords:

  • Glioma
  • Low-grade Glioma
  • RAD001
  • Glioma

Name

Location

Dana-Farber Cancer InstituteBoston, Massachusetts  02115
Seattle Cancer Care AllianceSeattle, Washington  98109
New York UniversityNew York, New York  10016
Children's Healthcare of AtlantaAtlanta, Georgia  30342
The Children'S HospitalDenver, Colorado  80218
University of Florida College of MedicineGainesville, Florida  32610
John Hopkins Medical CenterBaltimore, Maryland  21287
Memorial Sloan-Kettering Cancer InstituteNew York, New York  10065
Phoenix Children's Hospital Center for Cancer and Blood DisordersPhoenix, Arizona  85016
Doernbecher Children's Hospital Oregon Health & Science UniversityPortland, Oregon  97239