Trials Under Way for Patients with Brain Cancer

The following clinical trials are currently recruiting patients with brain cancer for inclusion in several investigations. Each trial description includes the NLM Identifier to use as a reference with ClinicalTrials.gov.




Perifosine and Temsirolimus for Recurrent/Progressive Malignant Gliomas

This phase 2, parallel-group, open label study will assess the effectiveness of temsirolimus and perifosine combination therapy in treating recurrent or progressive brain tumors. Patients aged ?18 years who have received previous radiotherapy and tem­ozolomide for the treatment of malignant glioma and who have (1) a Karnofsky performance score of ?70, (2) a life expectancy >8 weeks, (3) normal organ and marrow function, and (4) adequate renal and liver function may enroll if other criteria are met. Patients are randomized to receive cytoreductive surgery or no procedure. After antiemetic prophylaxis, both groups will receive perifosine according to protocol.

Primary outcome measures include radiographic response and 6-month, progression-free survival. The secondary outcome measure is median overall survival, measured from the time the study drug is first administered for up to 48 months or until the patient dies.

This study is expected to enroll 17 patients in New York City. For more information, contact Andrew B. Lassman, MD, at 212-342-0571 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT02238496.

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Valproic Acid, Radiation, and Bevacizumab in Children

This phase 2, single-group, open-­label trial examines whether the combination of valproic acid and bevacizumab with surgery and radiation will reduce brain tumors more effectively and improve the possibility of cure. Patients 3 to 21 years of age with high-grade gliomas who have not received previous chemotherapy, radiation, biologic therapy, or bone marrow transplants, and who have a Karnofsky or Lansky performance score of ?50 within 2 weeks of study enrollment may participate if other criteria are met.

Patients will be encouraged to have the maximal surgical resection that can be performed safely before entering into the study. All patients will receive valproic acid in a dose-escalation sequence and bevacizumab intravenously every 2 weeks during the maintenance phase, for a maximum of 24 months. Radiation therapy will start within 30 days of the patient’s definitive surgical procedure. Primary outcome measures are event-free survival and valproic acid toxicity, assessed over a time frame of 24 months. T

his study is expected to enroll 56 patients in Oklahoma City, OK, and at various sites throughout Texas. For more information, contact Jack Su, MD, at 832-822-4306 or This email address is being protected from spambots. You need JavaScript enabled to view it., or Susan Blaney, MD, at 832-822-4586 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT00879437.

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Palbociclib Isethionate for Treatment of Younger Patients

The goal of this phase 1, open-label study is to characterize the side effects and best dose of palbociclib isethionate in treating younger patients with recurrent, progressive, or refractory brain tumors. Patients aged 4 to 21 years who received their last dose of myelosuppressive anticancer chemotherapy ?3 weeks, nitrosourea ?6 weeks, or biologic agent ?7 days before enrolling in the trial are eligible to participate if other criteria are met. All patients will receive the study drug daily on days 1 to 21. This treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity.

Primary outcomes are the maximum tolerated dose of palbociclib isethionate and the incidence of adverse events, both assessed for a time frame of 4 weeks to 1 month. Secondary outcome measures are objective responses (ie, partial and complete response), and individual and population pharmacokinetic parameters.

This study is expected to enroll 40 patients in several locations throughout the United States. For more information, contact Wint M. Swe, MBA, MBBS, at This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT02255461.

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Postapproval Study of NovoTTF-100A

The purpose of this phase 4, nonrandomized, parallel-assignment study is to confirm that the efficacy of the NovoTTF-100A System in patients with recurrent glioblastoma multiforme is comparable to that of basic standard-of-care (BSC) chemotherapy. Patients aged ?22 years with a Karnofsky performance score of ?70, a histologic diagnosis of glioblastoma multiforme, and who have received maximal, safe, surgical resection; radiation therapy; and concomitant and maintenance temozolomide may enroll if other criteria are met. Patients will receive either BSC or NovoTTF-100A monotherapy.

The primary outcome is the overall survival, measured 5 years from initiation of accrual. Secondary outcome measures include the change in neurocognitive function from baseline, genetic profiling of tumors and correlation with response to treatment, and the adverse event profile.

This study is expected to enroll 486 patients at sites throughout the United States. For more information, contact Ghazala Kabani at This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT01756729.

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Photodynamic Therapy for Brain Tumors

This phase 1, open-label study evaluates the maximum tolerated dose and preliminary effectiveness trends of photodynamic therapy (PDT) in children. Patients aged 6 months to 18 years with relapsed or refractory brain tumors and potentially resectable disease, and whose disease does not have a known curative therapy or therapy proven to prolong survival with an acceptable quality of life may enroll if other criteria are met. All patients will receive intravenous Photofrin in a dose-escalation manner approximately 24 hours before tumor resection surgery and PDT.

The primary outcome measure is the maximum tolerated dose of Photofrin in pediatric patients. Dose-limiting toxicities observed can include neurotoxicity, photosensitivity, ocular sensitivity, and any other toxicity ?grade 4 within the same period. The secondary outcome measure is brain tumor response occurring within 3 years of receiving PDT.

This trial is expected to enroll 24 patients in Wauwatosa, WI. For more information, contact Michael E. Kelly, MD, PhD, at 414-266-6471 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT01682746.

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Genetically Modified Neural Stem Cells, Flucytosine, and Leucovorin

The objective of this phase 1, open-label study is to determine the side effects and best dose of genetically modified neural stem cells (NSCs) and flucytosine in combination with leucovorin. Patients aged ?18 years with recurrent, high-grade gliomas may enroll if other criteria are met. All patients will receive NSCs intracranially on days 1 and 15. Flucytosine is given orally on days 4 to 10 and 18 to 24. Patients may be given leucovorin orally, depending on when they enter the study.

Primary outcome measures include the maximum tolerated dose and incidence of dose-limiting toxicities, the incidence of all adverse events and toxicities, and clinically significant allergic reactions to NSCs. Secondary outcome measures include T-cell responses, antibodies against NSCs, and pharmacokinetic parameters.

This trial is expected to enroll 24 patients in Duarte, CA. For more information, contact Alexandra Ching, NP, at 626-471-9393 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT02015819.

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Veliparib, Radiation Therapy, and Temozolomide for Younger Patients

This phase 1/2 trial studies the side effects and best dose of veliparib in younger patients when given with radiation therapy and temozolomide. Patients aged ?21 years who have newly diagnosed diffuse intrinsic pontine gliomas, have a Karnofsky or Lansky performance score of ?50 (for patients >16 years of age or ?16 years of age, respectively), and are treatment-naïve except for surgery and/or steroids may enroll if other criteria are met. All patients will receive a dose escalation of veliparib orally 5 days a week for 6 to 7 weeks. During maintenance therapy, all patients will receive veliparib and temozolomide on days 1 to 5. Treatment repeats every 28 days for up to 10 courses in the absence of disease progression or unacceptable toxicity.

Primary outcome measures are the maximum tolerated dose of veliparib, the feasibility of intrapatient dose escalation of temozolomide during maintenance therapy with veliparib, and overall survival. Secondary outcomes are progression-free survival, number of pseudoprogression cases, and the pharmacokinetic parameters of veliparib.

This study is expected to enroll 66 patients at multiple locations throughout the United States. For more information, contact Patricia A. Baxter at 832-824-4681 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT01514201.

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Selumetinib in Young Patients with Recurrent or Refractory Low-Grade Glioma

This phase 1/2, single-group, open-label trial evaluates the side effects and best dose of selumetinib and its efficacy in the treatment of recurrent or refractory low-grade glioma. Patients aged 3 to 21 years who have not received myelosuppressive anticancer chemotherapy for ?3 weeks or a nitrosourea for ?6 weeks before study registration may enroll if other criteria are met. All patients will receive selumetinib orally twice daily on days 1 to 28. Courses repeat every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.

Primary outcome measures are the maximum tolerated dose and recommended phase 2 dose of selumetinib (as determined by dose-limiting toxicities), the stratum-specific objective response rate sustained for 8 weeks, objective response, and disease stabilization rates. Secondary outcome measures include plasma drug concentrations and pharmacokinetic parameters, stratum-specific progression-free survival (PFS) distribution, the presence or absence of BRAF V600E mutations or BRAF KIAA1549 fusion, and PFS in patients with recurrent disease.

This trial is expected to enroll 135 patients in locations throughout the United States. For more information, contact Jason R. Fangusaro at 312-227-4846 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT01089101.

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Phase 1 Study of a Dendritic Cell Vaccine

This phase 1, parallel-assignment study tests the safety and effectiveness of a dendritic cell vaccine in patients with newly diagnosed or recurrent glioblastoma. Patients aged ?18 years who have had a complete resection of their tumor and have a Karnofsky performance score of ?70 may enroll if other criteria are met. Patients are randomized to receive the dendritic cell vaccination and either standard temozolomide chemotherapy and involved field radiation therapy, or, for patients who were previously treated with bevacizumab, an optional retreatment with this drug.

Primary outcome measures assess safety and tolerability, the number of serious adverse events, and treatment-related toxicities over the course of 1 year. Secondary outcome measures evaluate overall and progression-free survival, health-related quality-of-life parameters, the overall response rate, immune response, and tumor stem-cell antigen expression, all over the course of 2 years.

This trial is expected to enroll 40 patients in Los Angeles. For more information, contact Cherry Sanchez, RN, at 310-423-6839 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT02010606.

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Carboplatin and Bevacizumab

The objective of this phase 2, single-group, open-label trial is to determine whether the combination of bevacizumab and carboplatin can help control recurrent ependymoma. Patients aged ?18 years with adequate bone marrow, liver, and renal function, and a Karnofsky performance score of ?60 may enroll if other criteria are met. All patients will receive bevacizumab 10 mg/kg intravenously on days 1 and 15 of each 28-day cycle. Carboplatin will be given intravenously on day 1 of each 28-day cycle. Primary outcome measures are the number of patients with progression-free survival at 1 year posttreatment.

This study is expected to enroll 46 patients in Boston, New York City, and Houston, TX. For more information, contact Mark R. Gilbert, MD, BS, at 713-792-2883. The NLM Identifier is NCT01295944.

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