Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
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 2
Funding Agency/Sponsor
Institutional
Disease
Pediatric Cancer
Enrollment 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