Protocol Summary

Protocol No.: OCR39902

Sponsor Protocol No.: MCC20339; MCC20339; P0208692

Protocol Title.: Evolutionary Therapy for Rhabdomyosarcoma

Principal Investigator: Lagmay, Joanne

Objective: 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.

Phase: Phase II

Age Group: Both

Age: N/A - N/A

Gender: All

Scope: National

Treatment:

Experimental: Arm A - First Strike
Participants will receive 42 weeks of conventional doses of vinorelbine, actinomycin D and cyclophosphamide

Experimental: Arm B - Second Strike - Maintenance
Participants will receive conventional doses of Vincristine/Actinomycin D/Cyclophosphamide (VAC) until complete response (CR) for 12-42 weeks and then switch to up to 2 years of vinorelbine/oral cyclophoshamide

Experimental: Arm C - Adaptive Therapy
Therapy with VAC that starts and stops based on response, adaptive timing of therapy, with a prolonged time to progression rather than complete remission goal

Active Comparator: Arm - D Conventional Therapy
Participants will receive a chemotherapy combination based on published trials. An example would be 42 weeks of VAC but may also include irinotecan, doxorubicin, ifosfamide, etoposide.

Detailed Eligibility:

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

Applicable Conditions:

  • Pediatric (Childhood) Cancer
  • Sarcoma
  • Participation Institution:

  • UF Gainesville : Ashley Bayne
  • Contact:
    Ashley Bayne
    Phone: +1 352-294-8745
    Email: abayne@ufl.edu

    More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT04388839