Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
About
A Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
This phase 3 trial aims to maintain excellent outcomes in patients with very low-risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study is to find out how well patients with low-risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.
Study Plan: This study wants to compare failure free survival (FFS) of patients with very low-risk rhabdomyosarcoma (VLR-RMS) when treated with vincristine and dactinomycin and evaluate the FFS of low-risk rhabdomyosarcoma (LR-RMS) when treated with vincristine, dactinomycin and cyclophosphamide
Arm VA (Very Low Risk): Patients with VLR-RMS receive vincristine intravenously (IV) on day 1 of each cycle and days 8 and 15 of cycles 1, 3, 5 and 7 and dactinomycin IV over 1 – 5 minutes or over 10 – 15 minutes on day 1 of each cycle. Treatment repeats every 21 days for 8 cycles in the absence of disease progression or unacceptable toxicity.
Arm VAC/VA (Low risk): Patients with LR RMS receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 1 – 3. Patients also receive dactinomycin IV over 1 – 5 minutes or 10 – 15 minutes and cyclophosphamide IV over 60 minutes on day 1 of each cycle. Treatment repeats every 21 days for 4 cycles in the absence of disease progression or unacceptable toxicity. Patients then receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 5 – 7 and dactinomycin IV over 1 – 5 minutes or over 10 – 15 minutes on day 1 of each cycle. Treatment repeats every 21 days for 4 cycles in the absence of disease progression or unacceptable toxicity. Patients may also undergo radiation therapy at cycle 5.
Arm M: Patients receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 2 – 4, 7 – 8, and 11 – 12 and dactinomycin IV over 1 – 5 minutes or 10 – 15 minutes on day 1 of cycles 2 – 5 and 8 – 14. Patients also receive cyclophosphamide IV over 60 minutes on day 1 of each cycle. Treatment repeats every 21 days for 12 – 13 cycles in the absence of disease progression or unacceptable toxicity. Patients may also undergo radiation therapy at cycle 5.
COG # ARST2032
Eligibility Criteria
Inclusion Criteria
- All patients must be enrolled on APEC14B1 and consented to the Molecular Characterization Initiative (Part A) prior to enrollment and treatment on ARST2032.
- Patients must be <= 21 years at the time of enrollment.
- Patients must have newly diagnosed embryonal rhabdomyosarcoma
- All patients will be evaluated for stage and clinical group. Note that clinical group designation assigned at the time of enrollment on study remains unchanged regardless of any second-look operation that may be performed.
- Patients will be eligible for the very low-risk stratum (Regimen VA) if they have Stage 1, CG I disease.
- Patients will be eligible for the low-risk stratum (Regimen VAC/VA) if they have Stage 1, CG II disease, Stage 2, CG I or II disease, or Stage 1, CG III (orbit only) disease.
- All patients will be evaluated for stage and clinical group. Note that clinical group designation assigned at the time of enrollment on study remains unchanged regardless of any second-look operation that may be performed.
- All patients and/or their parents or legal guardians must sign a written informed consent.
Exclusion Criteria
- Patients who have received prior chemotherapy and/or radiation therapy for cancer prior to enrollment. Surgical resection alone of previous cancer(s) is permitted.
- Patients who have received chemotherapy or radiation for non-malignant conditions (e.g., autoimmune diseases) are eligible. Patients must discontinue chemotherapy for non-malignant conditions prior to starting protocol therapy.
- Patients unable to undergo radiation therapy, if necessary, as specified in the protocol.
- Pregnant or breastfeeding females.
Primary Investigator
Glenn Edwards, MD, is the section chief of Pediatric Hematology/Oncology, as well as the Children's Oncology Group's principal investigator for Carilion Clinic. With over 30 years of clinical experience, he is board-certified by the American Board of Pediatrics in pediatrics and pediatric hematology/oncology. He completed his fellowship at Walter Reed Army Medical Center and completed his internship and residency at Tripler Army Medical Center.
Contact Information
Wendy McCarty, CCRP
Clinical Research Coordinator
Sydnee Moses, BS
Clinical Research Coordinator
