Phase I Cancer Studies

UPMC Children’s Hospital of Pittsburgh is proud to offer early phase clinical trials for patients with relapsed and refractory cancers through our participation in the Children’s Oncology Group Pediatric Early Phase Clinical Trial Network (PEP-CTN) and the Pediatric Brain Tumor Consortium. Working collaboratively with government, the medical community, and the pharmaceutical industry, PEP-CTN prioritizes and streamlines development of innovative, targeted therapies for children with cancer. UPMC Children’s Hospital is one of 21 sites in the country participating in PEP-CTN trials. Similarly, we are one of 11 academic centers and children’s hospitals in the U.S. that make up the Pediatric Brain Tumor Consortium, which also pursues early phase research initiatives.

The development of new therapies goes through multiple phases of testing. Phase I trials are the earliest and 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.

At UPMC Children’s, quality of life, especially at this stage of treatment, is a top priority for 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, UPMC 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.

The tab below shows Pilot and Phase I studies being done at UPMC Children’s. These are also found within separate categories listed at the left. Physicians or parents/guardians of pediatric patients who might be eligible are encouraged to ask about enrollment. Contact information is provided in each study description. Thank you for exploring these opportunities and helping us find cures for childhood cancer.

Clinical Studies

Central Nervous System Immunotherapy Using MK-3475 – Phase I

PBTC-045: Trial of MK-3475 in Children with Recurrent, Progressive, or Refractory Diffuse Intrinsic Pontine Glioma, Non-brainstem High-grade Gliomas, Ependymoma, Medulloblastoma, or Hypermutated Brain Tumors

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Central Nervous System Tumor Treatment with APX005M – Phase I

PBTC-051: Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma

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Central Nervous System Tumor Treatment with Savolitinib – Phase I

PBTC-049: Study of Savolitinib in Recurrent, Progressive, or Refractory Medulloblastoma, High-Grade Glioma, or Diffuse Intrinsic Pontine Glioma

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Chronic Myeloid Leukemia Treatment with Bosutinib – Phase I/II

AAML1921: A Study of a Drug, Bosutinib, for Treating Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph+ Chronic Myeloid Leukemia

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DIPG Treatment with Panobinostat – Phase I

PBTC-047: Panobinostat in Children with Diffuse Intrinsic Pontine Glioma

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Ependymoma Vaccine Trial – Phase I

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

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Glioma (High-Grade and DIPG) Therapy with Selinexor and Radiation – Phase I/II

ACNS1821: A Study of the Drug Selinexor with Radiation Therapy in Patients with Newly Diagnosed DIPG and High-Grade Glioma

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Glioma Treatment with AZD6244 – Phase I/II

PBTC-029B: Study of AZD6244 for Recurrent or Refractory Pediatric Low-Grade Glioma

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M0 Medulloblastoma Treatment in Very Young Children – Phase I

ADVL1314: Study of Eribulin Mesylate, a Novel Microtubule Targeting Chemotherapeutic Agent, in Children with Refractory or Recurrent Solid Tumors including Lymphomas

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Melanoma Treatment with Combination Binimetinib & Encorafenib – Phase Ib

Study of the Combination of Binimetinib and Encorafenib in Adolescent Patients with Unresectable or Metastatic BRAF V600-mutant Melanoma

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SHH Medulloblastoma Treatment with CX-4945 – Phase I/II

PBTC-053: Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma

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Solid Tumor and Lymphoma Treatment with PI3K – Phase I/II

ADVL1721: Study of PI3K Inhibitor Copanlisib in Pediatric Patients with Relapsed/Refractory Solid Tumors or Lymphoma

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Solid Tumor Treatment with Palbociclib – Phase I

ADVL1921: Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (IBRANCE®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors

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For More Information

Our clinical trial options vary frequently. For more information, please call 412-692-8864 and ask for a member of the Phase I or Neuro-Oncology program teams. Learn more about clinical studies, including frequently asked questions and how to participate.