Study of Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
Phase: Recruiting
Condition(s): Rhabdomyosarcoma
NCT Number: NCT05304585 Other Study ID Number(s): ARST2032
What Is the Purpose of This Study?
This phase III 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.
Who Can Take Part in This Study?
To be eligible for this study:
- All patients must be enrolled on APEC14B1 (NCT02402244) and consented to the Molecular Characterization Initiative (Part A) prior to enrollment and treatment on this trial.
- Patients must be =< 21 years at the time of enrollment.
- Patients must have newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma (ARMS).
What Will Happen During This Study?
Patients are assigned to 1 of 2 regimens based on clinical features. Patients with positive mutation status are transitioned to a third regimen, Regimen M.
Regimen VA:
- 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.
- Patients with MYOD1 or TP53 mutated tumors transition to Regimen M at cycle 2 (if mutation status is determined to be positive at week 3) or cycle 3 (if mutation status is determined to be positive after week 3).
Regimen VAC/VA:
- 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 with MYOD1 or TP53 mutated tumors transition to Regimen M at cycle 2 (if mutation status is determined to be positive at week 3) or cycle 3 (if mutation status is determined to be positive after week 3).
- Patients may also undergo radiation therapy at cycle 5.
Regimen 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.
Principal Investigator
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