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Trials in Progress

(P 551) PHASE II TRIAL OF GEMCITABINE AND NAB-PACLITAXEL IN PATIENTS WITH RECURRENT RHABDOMYOSARCOMA: A CLINICAL TRIAL IN PROGRESS FROM THE NATIONAL PEDIATRIC CANCER FOUNDATION

Location: The Liffey, Level 1

    Author(s)

  • Aditi Dhir, MD (she/her/hers)

    Assistant Professor, Pediatric Hematology Oncology
    University of Miami Miller School of Medicine
    Miami, Florida, United States

    • Co-Author(s)

  • JO

    Javier Oesterheld, MD

    Division Chief - LCH Cancer and Blood Disorders
    Atrium Health Levine Children's Hospital
    Charlotte, North Carolina, United States

  • PT

    Patrick Thompson, MD

    University of North Carolina Health Care
    Chapel Hill, North Carolina, United States

  • TB

    Thomas Badgett, MD

    University of Kentucky College of Medicine
    Lexington, Kentucky, United States

  • CP

    Christine A. Pratilas, MD

    Dr.
    Johns Hopkins University
    Baltimore, Maryland, United States

  • BS

    Bhuvana Setty, MD

    Associate Professor of Pediatrics
    Nationwide Children's Hospital
    Columbus, Ohio, United States

  • Masanori Hayashi, MD

    Assistant Professor
    Children's Hospital Colorado
    Aurora, Colorado, United States

  • Matteo Trucco, MD

    Associate Professor
    Cleveland Clinic
    Cleveland, Ohio, United States

  • Jonathan Metts, MD

    Pediatric Oncology
    Moffitt Cancer Center and Johns Hopkins All Children's Hospital
    Tampa, Florida, United States

  • DR

    Damon Reed, MD

    Moffitt Cancer Center
    Tampa, Florida, United States

    • Poster Presenter(s)

  • LW

    Lars M. Wagner, N/A, MD

    Duke Children's Hospital
    Durham, North Carolina, United States

Objective: The combination of gemcitabine and docetaxel is commonly used in patients with recurrent bone or soft tissue sarcoma. Nab-paclitaxel is a nanoparticle albumin-bound taxane with less myelosuppression than docetaxel, and preclinical studies in pediatric sarcoma models have demonstrated activity in models of rhabdomyosarcoma (RMS). The combination of gemcitabine and nab-paclitaxel is now frontline therapy for pancreatic cancer, and responses in RMS patients have been reported with this combination in small retrospective series (Metts et al, Pediatr Blood Cancer 2017; Tian et al, BMC Cancer 2020). Therefore, we are currently conducting a prospective multi-institutional phase II trial to better define the activity of this combination in patients with recurrent RMS (clinicaltrials.gov identifier NCT02945800). The study is currently open at 14 pediatric oncology centers in the United States through the National Pediatric Cancer Foundation.

Methods: Patients age > or equal to 3 years with relapsed or refractory RMS who have measurable disease and adequate organ function are eligible. The trial uses a single arm, Simon’s two-stage design to identify an objective response rate of > 35%. Patients receive weekly gemcitabine 1000 mg/m2 and nab-paclitaxel 125 mg/m2 x 3 in four-week cycles, using the schedule approved for pancreatic cancer. Pegfilgrastim is used for all patients. The protocol was amended to specify that if myelosuppression is limiting after dose reduction, an alternate schedule may be used in which both drugs are administered weekly x 2 in three-week cycles. Responses are assessed by RECIST version 1.1. Correlative studies include immunohistochemical assessment of potential biomarkers of chemosensitivity (SPARC, CAV-1, hENT1), radiomic analysis of imaging studies, and identification of circulating tumor cells and tumor DNA in peripheral blood.

Results: N/A ......

Conclusion: N/A ......