Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00901849
• Date of registration: 13/05/2009
• Prospective Registration: No
• Primary sponsor: Roger Packer
• Public title: Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)
• Scientific title: Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)
• Date of first enrolment: May 2007
• Target sample size: 21
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00901849
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion 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).



Age minimum: N/A
Age maximum: 21 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Low-grade Gliomas

Intervention(s)

Drug: Tarceva and Rapamycin

Primary Outcome(s)

Participants will remain on the trial for one year if benefiting from drug as demonstrated by radiographic evaluation and clinical evaluation. [Time Frame: one year]

Secondary ID(s)

4104

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Children's Research Institute