Brain Tumor
A Phase I Trial of Lenalidomide and Radiotherapy in Children With Diffuse Intrinsic Pontine Gliomas and High-grade Gliomas
NCI-10-C-0219, NCT01226940
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Investigator(s): |
Katherine E. Warren, M.D.
Principal Investigator
Phone: 301-496-4256
Fax: 301-480-2308
warrenk@mail.nih.gov
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Linda Ellison, R.N.
Research Nurse
Phone: 301-496-8009
Fax: 301-480-8871
ellisonl@mail.nih.gov
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Pediatric Oncology
Phone: 301-496-4256
1-877-624-4878 (Toll free)
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Background:
- Brain tumors are the most common solid neoplasm among children and the second most common group of pediatric cancers
- Standard therapeutic options for brain tumors consist of surgical resection, radiation therapy, and chemotherapy, yet overall survival rates for malignant brain tumors remain low
- Radiation therapy plays a key role in the treatment of diffuse intrinsic pontine gliomas (DIPG) and high-grade gliomas (HGG), and thalidomide has been shown in an animal model to be a radiosensitizer
- Lenalidomide, a thalidomide analog with antiangiogenic, cytotoxic, and immunomodulatory effects, is being evaluated for the treatment of central nervous system (CNS) tumors and has shown tolerability and activity in pediatric studies
Objectives:
- Determine the tolerability and toxicity profile of oral lenalidomide when administered to children with newly diagnosed high-grade gliomas (HGG) and diffuse intrinsic pontine gliomas (DIPG) with concurrent radiation at doses up to 116 mg/m2/day
- Evaluate long-term tolerability of lenalidomide in children with newly-diagnosed HGG and DIPG
- Evaluate magnetic resonance imaging (MRI) sequences for noninvasive monitoring of metabolic and biologic changes in malignant brain tumors with therapy
- Estimate 6-month and 12-month progression free survival (PFS) and overall survival (OS) in this patient population
- Determine if angiogenic and/or immunomodulatory biomarkers in the blood and urine correlate with toxicity and disease response
- Determine the rate of central nervous system (CNS) metastatic disease in patients treated with antiangiogenic chemotherapy
- Determine any correlation of steady state pharmacokinetics of lenalidomide with time to progression, number and type of toxicities, and dose limiting toxicities
Key Eligibility Criteria:
- Children with newly diagnosed high-grade gliomas (HGG) or diffuse intrinsic pontine gliomas (DIPG)
- < 19 years of age
- No prior chemotherapy or radiation therapy
- Performance score ≥ 60%
- Children with HGG must have an inoperable or incompletely resected tumor
- Clinical laboratory tests must be within stated limits
Study Outline:
- There will be two phases to therapy on this study—the radiation phase and the maintenance phase; the maximum tolerated dose (MTD) will be determined by tolerability during the radiation phase
- Eligible patients will receive radiation therapy 5 days per week to a prescription dose of 54-59.4 Gy, with concurrent administration of lenalidomide daily in a standard Phase 1 dose escalation design
- At the conclusion of radiation therapy, there will be a 2-week break, followed by maintenance dosing of lenalidomide for 21 days of a 28-day course, until unacceptable toxicity, disease progression, or completion of 24 courses of lenalidomide beyond radiotherapy
- Using a multi-center, Phase I dose-escalation design, the total sample size and the study duration are expected to be 18–30 patients and 5–6 years, respectively
Additional Information:
- 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.
- FAQs about this study - provides information for patients about the trial such as frequency and duration of visits, costs, how to enroll, and study outline.
- PDQ (Physicians Data Query) - provides additional details about this study for health care providers.
Reviewed: 12/14/12
Updated: 8/1/12
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