Evolutionary Therapy for Rhabdomyosarcoma

Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Sponsor score: 40
Inactive

ClinicalTrials ID:NCT04388839

Patient View

This trial assesses four chemotherapy strategies for newly diagnosed metastatic Fusion Positive Rhabdomyosarcoma. Eligible participants must have confirmed PAX/FOXO1 fusion and distant metastatic disease. Exclusions include regional lymph nodes as the sole disease site and uncontrolled illnesses. Interventions include Vincristine, Cyclophosphamide, Vinorelbine, and Actinomycin D.

Patient Parameters

Program Overview

This clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic Fusion Positive (alveolar) Rhabdomyosarcoma. The participant and their physician will choose from: Arm A) a first strike therapy, Arm B) a first strike-second strike (maintenance) therapy, Arm C) an adaptively timed therapy, and Arm D) conventional chemotherapy.

Eligibility Criteria

Inclusion Criteria

  • Participants must have a new histologic diagnosis of rhabdomyosarcoma
  • Participants must have FISH, PCR or other molecular confirmation of PAX/FOXO1 fusion per institutional standards
  • Participants must have sufficient tissue (up to 10 unstained FFPE) for correlative testing
  • All participants must have distant metastatic disease; either biopsy positive or PET avid extranodal or distant nodal lesions determined by the investigator to be metastatic disease. Patients with a single distant metastatic site that has been excised prior to study entry are eligible
  • No prior systemic chemotherapy
  • Participants enrolled to Arm B, maintenance, must be able to take oral cyclophosphamide. Note: enteral administration of cyclophosphamide is allowable.
  • Males and females of reproductive potential may not participate unless they have agreed to the use of, at minimum, two methods of contraception during and after treatment or abstinence.
  • Women of childbearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
  • Men who are sexually active with women of child bearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
  • All patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document.

Exclusion Criteria

  • Participants with regional lymph nodes as the only site of disease are not eligible. Distant nodal sites alone are eligible

  • Participants who are receiving any other investigational agents for rhabdomyosarcoma are ineligible

  • Participants must not be receiving any additional medicines being given for the specific purpose of treating cancer. Alternative medications including, but not limited to cannabis based products would not be a reason for exclusion

  • Participants are ineligible if they have uncontrolled intercurrent illness including, but not limited to:

    • ongoing or active infection not expected to resolve with current antibiotic plan
    • cardiac arrhythmia
    • psychiatric illness/social situations that would limit compliance with study requirements

  • Patients who are pregnant or breastfeeding are not eligible because there is no available information regarding human fetal or teratogenic toxicities. Females of childbearing potential must have a negative serum or urine pregnancy test within 24 hours of starting protocol therapy.

  • Participants who are considered unable to comply with the safety monitoring requirements of the study are not eligible

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