Clinical trial NCT00901849: Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)
Official title: Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Low-grade Gliomas
Lead sponsor: Children's National Research Institute
Sponsor class: OTHER
Collaborators: name: Children's National Research Institute
First posted: 2009-05-14
Enrollment: 21
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the feasibility of combining two drugs, Tarceva (an anti-EGFR agent), and Rapamycin (an mTOR inhibitor), in children with progressive low-grade gliomas who have failed initial conventional treatment. In addition to evaluating the toxicity of this drug regimen, the potential efficacy of the regimen will be assessed.


Eligibility criteria: Inclusion Criteria:

* children less than 21 years of age, with or without neurofibromatosis, with recurrent low-grade gliomas.
* patients with or without NF1 must have failed some form of conventional therapy, radiotherapy, or chemotherapy (carboplatin/vincristine) to be eligible, including those treated wtih initial surgery. Patients do not have to have received radiotherapy to be eligible.
* children with all types of histologically proven low-grade gliomas will be eligible; re-operation at the time of recurrence is not mandatory for entry on study.
* patients with intrinsic brain lesions, believed neuroradiographically consistent with a low-grade glial tumor, with neurofibromatosis type 1 can be treated without histological confirmation.
* patients without neurofibromatosis type 1 must have histological confirmation of a low-grade glial tumor prior to entry on study.
* patients must have a Karnofsky score of greater than or equal to 50% for children greater than 10 years of age, or a Lansky score of greater than or equal to 50% for patients less than 10 years of age.
* patients who are unable to walk because of paralysis, but who are able to use a wheelchair, will be considered ambulatory for purposes of assessing performance score.
* patients must have a life expectancy of at least 12 weeks.
* patients must be able to swallow medication in tablet form.
* patients must have adequate organ function, including: peripheral ANC of greater than 1,000/microliters; a platelet count of greater than 100,000/microliters; hemoglobin of greater than 8 gms,dl (pRBC transfusions of allowed to maintain hemoglobin \> 8 g/dl)
* patients must have adequate renal function, which is defined as a normal serum creatinine for age
* patients must have adequate liver function, as defined as a total bilirubin or less than 1.5 times the upper limit of normal for age, and an SGPT (ALT) of less than 2.5 times the upper limit of normal for age
* patients must have had a MR scan within 3 weeks of starting treatment
* all patients, and/or their parents or legal guardian, must sign a recent informed consent
* all institutional, FDA, and NCI requirements for human study must be met.

Exclusion Criteria:

* patients must not have any other active tumors.
* pregnancy or breast feeding is an exclusion criteria, as the potential mutagenicity and cytotoxicity of these drugs in developing fetuses are unknown. A pregnancy test must be obtained in females who are postmenarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.
* patients with uncontrolled infection are excluded.
* patients who have previously received Tarceva or Rapamycin are excluded.
* patients on antiepileptics and/or corticosteroids are allowed on study as long as they have been on a stable or weaning dose for 7 days prior to study initiation (defined as first day of treatment).

Primary outcomes:
measure: Participants will remain on the trial for one year if benefiting from drug as demonstrated by radiographic evaluation and clinical evaluation.
timeFrame: one year
Secondary outcomes:
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's Research Institute
state: District of Columbia
Study officials:
References: