Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma

Conditions

• Embryonal Rhabdomyosarcoma
• Fusion-Negative Alveolar Rhabdomyosarcoma
• Spindle Cell/Sclerosing Rhabdomyosarcoma

Eligibility Criteria

Sex

ALL

Age

N/A - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Biopsy Procedure — PROCEDURE
  Undergo tumor biopsy
• Bone Scan — PROCEDURE
  Undergo bone scan
• Computed Tomography — PROCEDURE
  Undergo CT scan
• Cyclophosphamide — DRUG
  Given IV
• Dactinomycin — BIOLOGICAL
  Given IV
• Magnetic Resonance Elastography — PROCEDURE
  Undergo MRI
• Positron Emission Tomography — PROCEDURE
  Undergo PET scan
• Radiation Therapy — RADIATION
  Undergo radiation
• Vincristine — DRUG
  Given IV

Study Details

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. 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. Another aim of the study it 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.

Key Dates

Start date

Aug 4, 2022

Status verified

Oct 2025

Primary completion

Jun 30, 2030

Completion

Jun 30, 2030

Study Design

Enrollment

205 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Regimen M (positive mutation)
  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. Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.
• Experimental: Regimen VA (VLR RMS)
  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). Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.
• Experimental: Regimen VAC/VA (VL RMS)
  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). Radiation therapy (if needed) will be administered at cycle 5.Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.

Primary Outcome Measure

Failure free survival (FFS) for very low risk patients [ Time Frame: From study enrollment to disease progression, recurrence, or death as a first event, assessed up to 3 years ]

Locations (165)

Find similar trials in Birmingham, AL

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  Recruiting · Children's Hospitals and Clinics of Minnesota · Minneapolis, Minnesota