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

Low-grade Gliomas Clinical Trial

Official title:

Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00901849
• Other study ID #: 4104
• Status: Completed
• Phase: Phase 1
• First received: May 13, 2009
• Last updated: September 12, 2014
• Start date: May 2007
• Est. completion date: October 2012

Study information

• Verified date: September 2014
• Source: Children's Research Institute
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: October 2012
• Est. primary completion date: July 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 21 Years

Eligibility

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).

Study Design

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

Related Conditions & MeSH terms

• Glioma
• Low-grade Gliomas
• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• Tarceva and Rapamycin
Tarceva will be administered once a day for 28 days without interruption. THen Rapamycin will be administered, orally twice daily, in combination with the Tarceva for the remainder of the study.

Locations

Country	Name	City	State
United States	Children's Research Institute	Washington	District of Columbia

Sponsors (2)

Lead Sponsor	Collaborator
Roger Packer	Children's Research Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Participants will remain on the trial for one year if benefiting from drug as demonstrated by radiographic evaluation and clinical evaluation.		one year	Yes