Hematology/Oncology Clinical Studies

Through clinical trials, the Pediatric Hematology/Oncology team at Children's Hospital of Pittsburgh of UPMC is accelerating the search for cures to childhood cancer. Collaborating closely with the Children’s Oncology Group (COG), the only pediatric oncology clinical trials group funded by the National Cancer Institute and National Institutes of Health, Children’s is among an elite group of hospitals participating in Phase I clinical trials through COG and the Pediatric Brain Tumor Consortium.

To help you locate a study relevant to your interests, we have organized them below.

Simply select a category and then browse the studies within the category to review brief descriptions and requirements. Or if you are looking for a study dealing with a specific condition, please use the search tool at the top of this page. Be sure to include the word "study" along with the name of the condition. Physicians or parents/guardians of pediatric patients who might be eligible are encouraged to inquire about enrollment. Contact information is listed at the end of each study.

Phase I Cancer Studies

Children’s Hospital of Pittsburgh of UPMC is one of only 21 hospitals qualified to be part of the Children’s Oncology Group’s elite Phase I/Pilot Research Consortium.

Phase I drug studies are the most experimental in nature, and therefore are conducted with the smallest groups of volunteers. They are initiated after drugs have passed animal and cell studies, and are offered to those children for whom standard care and other clinical studies have already failed. Although Phase I experimental therapeutics present hope for such situations, as with any experimental treatment, there are no guarantees.

Quality of life, especially at this stage of treatment, is a top priority for Children’s medical and research team members, who are acutely tuned to the needs of patients and their families. Families who come from outside the Greater Pittsburgh region may incur travel costs. For Phase I studies in particular, Children’s can help through support it receives from Alex’s Lemonade Stand Foundation. Lodging for family members may be provided through the Ronald McDonald House.

Children’s studies in hematology and oncology are organized according to the seven groups listed below, and Pilot and Phase I clinical trials are labeled within each grouping for easy identification.

Simply select a category and then browse the studies within the category to review brief descriptions and requirements. Physicians or parents/guardians of pediatric patients who might be eligible are encouraged to inquire about enrollment. Contact information is listed at the end of each study.

Thank you for exploring these opportunities and helping us to find cures and prevent childhood disease.

Clinical Studies


Acute Lymphoblastic Leukemia and B-Lineage Lymphoma Treatment via Risk-Adapted Chemotherapy – Phase III

Risk-Adapted Chemotherapy in Treating Younger Patients with Newly Diagnosed Standard-Risk B-Acute Lymphoblastic Leukemia or localized B-Lineage Lymphoblastic Lymphoma Read more

Acute Lymphoblastic Leukemia Cell Banking Study

Proper Protocols for Collecting and Banking Relapsed Acute Lymphoblastic Leukemia Research Specimens Read more

Acute Myeloid Leukemia trial using Bortezomib and Sorafenib – Phase III

Trial for Patients with de novo Acute Myeloid Leukemia using Bortezomib and Sorafenib for Patients with High Allelic Ratio FLT3/ITD Read more

Brain Tumor Memory Study – Phase II

Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Brain Tumor Read more

Central Nervous System Tumor Treatment with PD-0332991 – Phase I

A Study of CDK 4-6 Inhibitor PD-0332991 in Children with Recurrent, Progressive or Refractory Central Nervous System Tumors Read more

Craniopharyngioma Treatment with PEGIntron – Phase II

Study of Peginterferon alfa-2b (PEGIntron) for Pediatric Patients with Unresectable or Recurrent Craniopharyngioma Read more

DIPG Treatment with MK-1775 and Local Radiation – Phase I

MK-1775 and Local Radiation Therapy in Treating Younger Patients With Newly Diagnosed Diffuse Intrinsic Pontine Gliomas Read more

Ependymoma Treatment with Maintenance Chemotherapy – Phase III

Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Read more

Ependymoma Vaccine Trial – Pilot

Treatment for Recurrent Ependymomas in Children Using HLA-A2 Restricted Tumor Antigen Peptides in Combination With Imiquimod Read more

Ewing Sarcoma Tissue Banking Study

A Children’s Oncology Group Protocol for Collecting and Banking Ewing Sarcoma Specimens Read more

Glioma Treatment with Selumetinib (AZD6244) – Phase II

Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma Read more

Glioma Treatment with Veliparib, Radiation and Temozolomide – Phase I/II

ABT-888, an Oral Poly (ADP-ribose) Polymerase Inhibitor, and Concurrent Radiation Therapy, Followed by ABT-888 and Temozolomide, in Children with Newly Diagnosed Diffuse Pontine Gliomas Read more

Gliomas Vaccine Study – Pilot

Treatment of Newly Diagnosed Brainstem Gliomas and Non-Brainstem High-Grade Gliomas, Recurrent Low-Grade and High-Grade Gliomas Read more

High-Risk Acute Lymphoblastic Leukemia Treatment with Combination Chemotherapy – Phase III

Trial for Newly Diagnosed High Risk B-precursor Acute Lymphoblastic Leukemia, Testing Clofarabine in the Very High Risk Stratum Read more

Hodgkin Disease Banking Study

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Hodgkin Lymphoma Treatment with Brentuximab Vendotin and Gemcitabine – Phase I/II

Brentuximab Vedotin in Combination with Gemcitabine for Pediatric and Young Adult Patients with Relapsed or Refractory Hodgkin Lymphoma Read more

Medulloblastoma and PNET Therapies with Temozolomide, Irinotecan, and Bevacizumab – Phase II

Temozolomide with Irinotecan versus Temozolomide, Irinotecan plus Bevacizumab for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial Read more

Medulloblastoma Treatment with Carboplatin and Isotretinoin – Phase III

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients Read more

M0 Medulloblastoma Treatment in Very Young Children – Phase I

Study of Eribulin Mesylate, a Novel Microtubule Targeting Chemotherapeutic Agent, in Children with Refractory or Recurrent Solid Tumors including Lymphomas Read more

M0 Medulloblastoma Treatment in Young Children – Phase II

Study for the Treatment of Non-Metastatic Nodular Desmoplastic Medulloblastoma in Children Less Than 4 Years of Age Read more

Neuroblastoma Biology Studies

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Osteosarcoma Tissue Banking Study

A Children’s Oncology Group Protocol for Collecting and Banking Osteosarcoma Specimens Read more

Pediatric Thrombophilia Registry

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Pediatric Tumors and Specimen Study

A Children’s Oncology Group Protocol for Collecting and Banking Pediatric Specimens Including Rare Pediatric Tumors Read more

Plexiform Neurofibromas Treatment with PEG-Intron - Phase II

Peginterferon alfa-2b in Treating Young Patients with Unresectable, Sympotomatic or Life-Threatening Plexiform Neurofibromas Associated with Neurofibromatosis Type 1 Read more

Renal Tumors Classification, Biology and Banking Study

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Sickle Cell Acute Chest Syndrome Treatment with Prasugrel – Phase III

A Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease Read more

Sickle Cell Disease Treatment with BMT and Conditioning (STRIDE) – Phase II

A Study of Hematopoietic Stem Cell Therapy for Young Adults with Severe Sickle Cell Disease Read more

Sickle Cell Medication Management Study

Patient-Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy with Hydroxyurea in Patients with Sickle Cell Disease Read more

Sickle Cell Pain Crisis Management Study (SUSTAIN) – Phase II

Study to Assess Safety and Efficacy of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises (SUSTAIN) Read more

Sickle Cell Vaso-Occlusive Pain Crisis Treatment Study (EPIC) – Phase II

Evaluation of Purified Poloxamer 188 (MST 188) in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC) Read more

Solid Tumor and Lymphoma Treatment with PF-02341066 – Phases I and II

PF-02341066, an Oral Small Molecule Inhibitor of Anaplastic Lymphoma Kinase (ALK) and c-MET, in Children with Relapsed/Refractory Solid Tumors and Anaplastic Large Cell Lymphoma Read more

Solid Tumor or ALCL Treatment with Crizotinib and Conventional Chemotherapy – Phase I

Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma Read more

T-cell Lymphoblastic Leukemia and Lymphoma Treatment with Bortezomib – Phase III

Bortezomib on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy) Read more

Transient Myeloproliferative Disorder Blood Study

Biology Study of Transient Myeloproliferative Disorder in Children with Down Syndrome Read more

Veno-Occlusive Disease Treatment with Defibrotide

Defibrotide for Treatment of Veno-Occlusive Disease of the Liver Read more

Wilms Tumor Treatment with Combination Chemotherapy and Surgery – Phase III

Treatment for Patients with Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor Read more

Linda McAllister, MD, PhD

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Cheryl Hillery, MD

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Peter Shaw, MD

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Debra Cohen, MD

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James Cooper, MD

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Melvin Deutsch, MD

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Erika Friehling, MD

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James Graves, PhD, MD

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Lynn Malec, MD

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Gary Mason, MD

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Scott Maurer, MD

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Linda McAllister, MD, PhD

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Amma Owusu-Ansah, MD

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Edward Prochownik, MD, PhD

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A. Ritchey, MD

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Peter Shaw, MD

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Jean Tersak, MD

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Randy Windreich, MD

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Michael Wollman, MD

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