Chemotherapy for Rhabdomyosarcoma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing four different chemotherapy schedules for patients with a specific type of advanced cancer. The goal is to find the best way to treat the disease by comparing different timing and combinations of chemotherapy treatments.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications, but you cannot be on any other cancer treatments. Alternative medications like cannabis products are allowed.
Is chemotherapy for rhabdomyosarcoma generally safe in humans?
Vinorelbine, a drug used in chemotherapy, has been studied for safety and shows a favorable safety profile with manageable side effects like neutropenia (low white blood cell count) and limited neurotoxicity. It has been tested in various cancers, including lung and breast cancer, and can be administered safely in outpatient settings.12345
What makes the chemotherapy treatment for rhabdomyosarcoma unique?
This chemotherapy treatment for rhabdomyosarcoma is unique because it combines vinorelbine with low-dose cyclophosphamide, which has shown promise as a maintenance regimen for high-risk patients. Vinorelbine, a semisynthetic vinca alkaloid, has demonstrated effectiveness in relapsed cases, particularly in alveolar rhabdomyosarcoma, and is used alongside other drugs like actinomycin-D and vincristine to enhance treatment outcomes.678910
What evidence supports the effectiveness of the drugs used in the chemotherapy for rhabdomyosarcoma?
Research shows that vinorelbine, when combined with cyclophosphamide, has shown clinical activity in treating relapsed rhabdomyosarcoma. Additionally, a novel regimen including vinorelbine, ifosfamide, vincristine, and actinomycin-D demonstrated a major response in high-risk rhabdomyosarcoma patients.6781011
Who Is on the Research Team?
Jonathan Metts, MD
Principal Investigator
Moffitt Cancer Center
Are You a Good Fit for This Trial?
This trial is for individuals newly diagnosed with metastatic Fusion Positive Rhabdomyosarcoma. They must have tissue available for testing, no prior chemotherapy, and molecular confirmation of PAX/FOXO1 fusion. Participants need to agree to contraception use if applicable and be able to take oral medications if in Arm B. Pregnant or breastfeeding women are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive chemotherapy based on chosen strategy: first strike, first strike-second strike (maintenance), adaptively timed, or conventional chemotherapy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Actinomycin D
- Cyclophosphamide
- Cyclophosphamide Pill
- Vincristine
- Vinorelbine
Actinomycin D is already approved in European Union, United States, Canada for the following indications:
- Rhabdomyosarcoma
- Wilms tumor
- Ewing's sarcoma
- Rhabdomyosarcoma
- Wilms tumor
- Ewing's sarcoma
- Trophoblastic neoplasm
- Rhabdomyosarcoma
- Wilms tumor
- Ewing's sarcoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
H. Lee Moffitt Cancer Center and Research Institute
Lead Sponsor
National Pediatric Cancer Foundation
Collaborator