Clinical Trials Under Way for Pediatric, Adolescent, and Young Adult Patients with Cancer

The following clinical trials are currently recruiting pediatric, adolescent, and young adult patients with cancer for inclusion in several investigations targeting blood and brain cancers in this population. Each trial description includes the NLM Identifier to use as reference with ClinicalTrials.gov.


1. Autologous T-Lymphocytes Genetically Targeted to the B-Cell Specific Antigen CD19 in Patients with Relapsed B-Cell Acute Lymphoblastic Leukemia
This phase 1 study will test the safety of giving pediatric and young adult patients with relapsed B-cell acute lymphoblastic leukemia special cells made from their own blood called “modified T-cells.” The goal of the study is to find a safe dose of these modified T-cells for patients whose leukemia has returned to the bone marrow.

To be eligible for infusion of modified T-cells during the treatment arm of this study, patients must be younger than 26 years of age and have a history of relapsed/refractory CD19-specific B-cell acute lymphoblastic leukemia involving the marrow. It is important for physicians to note that on this particular protocol, ?25% blasts by morphology and/or flow cytometry constitute a bone marrow relapse. In addition, the patients must have experienced ?2 relapses, with an early first marrow relapse after complete remission (CR) at <18 months. Additionally, an intermediate/late first marrow relapse (first CR >18 months from first CR) with poor initial response (?5% blasts by morphology and/or flow cytometry) following reinduction chemotherapy also constitutes a bone marrow relapse.
For additional inclusion and exclusion criteria, contact the principal investigator, Kevin Curran, MD, at 212-639-5836. The trial is being conducted at Memorial Sloan-Kettering Cancer Center, New York, New York. The NLM Identifier is NCT01860937. Source: ClinicalTrials.gov, October 17, 2013.

2. GNKG168 in Patients with Acute Lymphoblastic Leukemia and Acute Myelogenous Leukemia
GNKG168, an investigational drug, is a Toll-like receptor (TLR) agonist, which is a novel approach to stimulating an effective anti­­-
tumor immune response because they are able to stimulate both innate and adaptive immune responses. In this phase 1 trial, GNKG168 will be studied in patients with relapsed and refractory acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML) who are younger than 21 years of age. Patients in the study would be in morphologic remission but be positive for minimum residual disease.

There will be 2 strata: patients who have received hematopoietic stem cell transplant and patients who have not. GNKG168 will be administered as a 60-minute intravenous infusion. One 14-day cycle consists of a 5-day treatment period followed by a 9-day rest period, and patients will receive 2 cycles before evaluation. The primary objective of the study is to determine the maximum tolerated dose of GNKG168 in relapsed ALL and AML patients.

For inclusion into the study, pa­tients must be ?1 and ?21 years of age when originally diagnosed with ALL or AML, and they must have previously histologically confirmed ALL or AML at their original diagnosis or previous relapse. Additionally, the patients must be in complete remission with less than 5% blasts in the bone marrow.

The trial is being conducted by the Therapeutic Advances in Childhood Leukemia Consortium. Contact Jeannette van der Giessen at 323-361-8725 for more information, including inclusion/exclusion criteria and study sites. The NLM Identifier is NCT01743807. Source: ClinicalTrials.gov, October 17, 2013.

3. Safety Study of CPX-351 in Children with Relapsed Acute Myeloid Leukemia (AML)
In this phase 1 study of children and adolescents 1 to 30 years of age, a drug called CPX-351 will be tested in different doses to assess the drug’s pharmacokinetics, toxicity, and tolerability in pediatric and young adults with relapsed/refractory hematologic malignancies to see which dose is safer in the study population.

CPX-351 has not yet been approved by the US Food and Drug Administration; it has only been used in research studies. The drug is made up of 2 chemotherapy drugs that patients may have already received, cytarabine and daunorubicin, which are now packaged together. The drug has previously been tested in adults. Pediatric and young adult patients with blood cancers such as leukemia and lymphoma are being recruited for the study. These patients must have already been treated with standard chemotherapy, but have disease that is still growing or has come back.

The subjects will receive a single course of CPX-351 administered on days 1, 3, and 5. The study will first be open to children in a dose-exploration phase, and then be available to an expanded cohort, which will be open to children and young adults once a tolerable dose has been determined.

Also in this study, researchers will collect blood samples for special pharmacokinetic studies that will measure how much CPX-351 remains in the body over time. These studies require the collection of several blood samples before and after the participants are given the study drug.

The single-institution study is conducted at Cincinnati Children’s Hospital Medical Center, Cincinnati, OH. Celator Pharmaceuticals, Inc is the study sponsor. For more information, including full inclusion/exclusion criteria, contact Laura Fossett, MS, at 513-636-2799. The NLM Iden­tifier is NCT01943682. Source: Clinical Trials.gov, October 17, 2013.

4. Fixed versus Flexible Filgrastim to Accelerate Bone Marrow Re­­­­covery after Chemotherapy
This randomized phase 3 trial will study the flexible administration of filgrastim after combination chemo­therapy to see how well it works compared with the fixed administration of filgrastim in decreasing the side effects of chemotherapy in younger patients (1 to 25 years of age) with cancer.

Because filgrastim is customarily used on a fixed schedule starting early after initiation of chemotherapy and there are data that these early doses may not be needed, this study is testing a new flexible schedule of filgrastim with the goal to optimize its use by reducing the number of painful shots, the cost of treatment, and the drug’s side effects in children with cancer receiving chemotherapy.

The study is being conducted at the Barbara Ann Karmanos Cancer Institute, Detroit, MI, by the National Cancer Institute. For more information, including full inclusion and exclusion criteria, contact Maxim Y. Yankelevich at 313-745-5515 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The NLM Identifier is NCT01987596. Source: Clinical Trials.gov, Novem­ber 26, 2013.

5. Veliparib, Radiation Therapy, and Temozolomide in Treating Younger Patients with Newly Diagnosed Diffuse Pontine Gliomas
This phase 2/3 trial is studying the side effects and optimal dose of veliparib given together with radiation therapy and temozolomide to see how well it works in treating younger patients (21 years of age and younger) who are newly diagnosed with diffuse pontine gliomas. Veliparib may stop the growth of tumor cells by blocking some of the enzymes that are needed for cell growth, and when used together with radiation therapy and chemotherapy, such as temozolomide, the drug may kill more tumor cells.

The study aims to identify the maximum tolerated dose or recommended phase 2 dose of veliparib that can be safely administered concurrently with radiation therapy, followed by maintenance therapy with veliparib and temozolomide. It will study the plasma pharmacokinetics of veliparib during combination veliparib and radiation therapy. It will also study the feasibility of intrapatient dose escalation of temozolomide during maintenance therapy with veliparib and temozolomide.

For inclusion into the study, patients with newly diagnosed diffuse intrinsic pontine gliomas, which are defined as tumors with a pontine epicenter and diffuse intrinsic involvement of the pons, are eligible without histologic confirmation. Patients with brain stem tumors that do not meet these criteria or with brain stem tumors that are not considered to be typical intrinsic pontine gliomas will only be eligible if the tumors are biopsied and proven to be an anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, or anaplastic mixed glioma. Patients with juvenile pilocytic astrocytoma, pilomyxoid astrocytoma, fibrillary astrocytoma, gangliogliomas, or other mixed gliomas without anaplasia are not eligible.

The study is being conducted by the National Cancer Institute. Patricia A. Baxter, Texas Children’s Hospital, is the principal investigator. She can be contacted by e-mail at This email address is being protected from spambots. You need JavaScript enabled to view it.. The study sites are in: Washington, DC; Chicago, IL; Bethesda, MD; Durham, NC; Philadelphia, PA; Pittsburgh, PA; Memphis, TN; and Houston, TX. The NLM Identifier is NCT01514201. Source: Clinical Trials.gov, October 17, 2013.

6. MK-1775 and Local Radiation Therapy in Treating Newly Diagnosed, Diffuse Intrinsic Pontine Gliomas
This phase 1 trial is studying the side effects and best dose of MK-1775, a WEE1 inhibitor, when given together with local radiation therapy to treat pediatric and young adult patients (37 months to 21 years of age) with newly diagnosed, diffuse intrinsic pontine gliomas (DIPGs). MK-1775 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The trial will also define and describe the toxicities of MK-1775 when given concurrently with radiation therapy in children with newly diagnosed DIPGs.

Patients with newly diagnosed DIPGs, which are defined as tumors with a pontine epicenter and diffuse involvement of the pons, are eligible without histologic confirmation. Patients with brain stem tumors that do not meet these criteria or are not considered to be typical intrinsic pontine gliomas will only be eligible if the tumors are biopsied and proven to be an anaplastic astrocytoma, glioblastoma, gliosarcoma, or anaplastic mixed glioma; patients with pilocytic astrocytoma, fibrillary astrocytoma, gangliogliomas, or other mixed gliomas without anaplasia are not eligible.

The National Cancer Institute is conducting the study. Sabine Mueller, MD, PhD, assistant professor of neurology, pediatrics, and neurosurgery at the University of California San Francisco School of Medicine, Department of Pediatrics, is the principal investigator. She can be contacted at 415-476-3831 or This email address is being protected from spambots. You need JavaScript enabled to view it.. The study sites are in: Arcadia, CA; Orange, CA; San Francisco, CA; Atlanta, GA; Indianapolis, IN; Minneapolis, MN; St. Louis, MO; Cincinnati, OH; Pittsburgh, PA; and Memphis, TN. The NLM Identifier is NCT01922076. Source: ClinicalTrials.gov, October 17, 2013.

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