Evolutionary Therapy for Rhabdomyosarcoma

Launched by H. LEE MOFFITT CANCER CENTER AND RESEARCH INSTITUTE · May 11, 2020

Keywords

ClinConnect Summary

This clinical trial, called "Evolutionary Therapy for Rhabdomyosarcoma," is exploring different ways to use chemotherapy for children and young adults who have just been diagnosed with a specific type of rhabdomyosarcoma known as metastatic Fusion Positive (alveolar) rhabdomyosarcoma. The trial is testing four different treatment plans: one involves an initial strong therapy, another combines an initial treatment with a follow-up maintenance therapy, a third uses an adaptable treatment schedule, and the last is a standard chemotherapy approach. The goal is to find out which method works best for patients.

To be eligible for this trial, participants must have a recent diagnosis of rhabdomyosarcoma that has spread to other parts of the body and must confirm a specific genetic marker associated with the disease. They should not have received any prior chemotherapy and must have enough tissue for testing. Participants of all genders and ages can join, but those who can become pregnant must agree to use contraception during and after the treatment period. If you or your loved one participate, you'll work closely with doctors to monitor health and treatment effects throughout the study. This trial is currently recruiting participants, so if you’re interested, it’s a good idea to discuss it with your healthcare team.

Gender

ALL

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