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Brain Tumor

NCI-12-C-0136, NCT01553149

Investigator(s):
Katherine E. Warren, M.D. Principal Investigator Phone: 301-496-4256 Fax: 301-480-2308 warrenk@mail.nih.gov
Referral Contact(s):
Linda Ellison, R.N. Research Nurse Phone: 301-496-8009 Fax: 301-480-8871 ellisonl@mail.nih.gov	Pediatric Oncology Phone: 301-496-4256 1-877-624-4878 (Toll free)

Given the poor outcome of patients with recurrent or refractory gliomas and the limited activity of most cytotoxic agents, new agents with novel mechanisms of action are needed
Inhibition of angiogenesis offers a new approach based upon the dependence of tumor growth on angiogenesis
Lenalidomide is an oral agent with both antiangiogenic and immunomodulatory activity that is well tolerated in adults and children and appears to have some activity in childhood CNS tumors

To determine the objective response rate of children with recurrent, refractory or progressive juvenile pilocytic astrocytomas (JPA) and optic pathway gliomas (OPG) who are treated with Regimen A low-dose (20 mg/m²/dose) or Regimen B high-dose (115 mg/m²/dose) lenalidomide

Estimate event-free survival (EFS) in children with recurrent, refractory, or progressive juvenile pilocytic astrocytomas and optic pathway gliomas who are treated with lenalidomide
Compare response categories and EFS across the 3 MR sequences (T2-weighted, FLAIR, T1-weighted post-contrast)
Correlate steady state pharmacokinetics of lenalidomide (1 sample obtained between Days 5 and 21) with objective response and EFS
Evaluate the hematologic toxicity of long term lenalidomide

< 22 years of age at the time of enrollment
BSA ≥ 0.4 m² at the time of enrollment
Diagnosis of pilocytic astrocytoma or optic pathway glioma that has relapsed, progressed, or become refractory to conventional therapy with measureable disease; patients with neurofibromatosis (NF-1) are eligible
Performance level ≥ 60 percent (Lansky or Karnofsky)
Histologic verification of the malignancy at original diagnosis or at the time of recurrence; histologic confirmation for patients with progressive optic pathway gliomas will not be required
Patients must have been treated with two or fewer anti-cancer regimens, including chemotherapy, biologic agents, immunotherapy, vaccines, monoclonal antibodies, or radiation therapy
At least one prior treatment regimen must have included carboplatin
Patients who have received prior radiation therapy for this tumor are eligible
Able to swallow intact capsules

This study is a randomized comparison of response rate among patients with recurrent, refractory, or progressive juvenile pilocytic astrocytomas and optic pathway gliomas who are treated with lenalidomide
Lenalidomide will be given either as a low-dose (20 mg/m²/dose) or high-dose (115 mg/m²/dose) regimen by mouth (PO) for 21 days of each 28 day cycle
Treatment will continue for a maximum of 26 cycles in the absence of disease progression or unacceptable side effects
Response will be evaluated prior to every other cycle for the first 13 cycles and prior to every third cycle for cycles 14 to 26; correlation of steady state pharmacokinetic levels with patient outcome will be performed
A total of 74 eligible patients will be enrolled and randomly assigned to either low-dose (Regimen A) or high-dose (Regimen B) arm; a maximum of 8 patients will be enrolled at NCI POB

This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
There is no charge for medical care received at NIH Clinical Center.

PDQ (Physicians Data Query) - provides additional details about this study for health care providers.

Reviewed: 12/14/12
Updated: 8/1/12