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ARST2031: A Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma

Title ARST2031: A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy with Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) plus VINO-CPO Maintenance in Patients with High Risk Rhabdomyosarcoma (HR-RMS)
Therapeutic Area Rhabdomyosarcoma (Tufts Children's)
Principal Investigator Jason Law, MD
Max Age 50 Years
Gender Any
Contact Sarah Sexton
More Information


This phase III trial compares the effect of vinorelbine with vincristine, dactinomycin, and cyclophosphamide (VAC) followed by vinorelbine and cyclophosphamide versus VAC followed by vinorelbine and cyclophosphamide for the treatment of high risk rhabdomyosarcoma.

Chemotherapy drugs, such as vinorelbine, vincristine, dactinomycin, and cyclophosphamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Giving vinorelbine and VAC may kill more tumor cells. Adding maintenance therapy (vinorelbine and cyclophosphamide) after VAC therapy, with or without vinorelbine, may help get rid of the cancer and/or lower the chance that the cancer comes back.

Study Details

Inclusion Criteria

  1. Patients with newly diagnosed RMS of any subtype, except adult-type pleomorphic, based upon institutional histopathologic classification are eligible to enroll on the study based upon Stage, Group, and age
  2. Adequate renal function defined as:
    • Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or
    • A serum creatinine based on age/gender
  3. Adequate liver function defined as:
    • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
    • If there is evidence of biliary obstruction by tumor, then total bilirubin must be < 3 x ULN for age.

Exclusion Criteria

  1. Patients with the following pre-existing conditions:
    1. Patients with evidence of uncontrolled infection.
    2. RMS that is considered a second malignancy and previous cancer(s) that were treated with chemotherapy and/or radiation. Surgical resection alone of previous cancer(s) is allowed.
    3. Patients with certain central nervous system involvement of RMS
  2. Patients who have received any chemotherapy (excluding steroids) and/or radiation therapy for RMS prior to enrollment (except patients requiring emergency radiation therapy for RMS.
  3. Patients who are pregnant or breastfeeding

Study Requirements

This study will be conducted in two consecutive phases: a safety and feasibility phase, in which 8 patients will be non-randomly assigned to receive VINO-AC, followed by a randomized efficacy phase.

In the efficacy phase, all patients will be randomized in a 1:1 ratio at study entry to receive VAC (Regimen A) or VINO-AC (Regimen B).

  • Patients randomized to the Regimen A will receive 42 weeks of VAC.
  • Patients randomized to Regimen B will receive 42 weeks of VINO-AC with vincristine on the third week (Day 15) of each cycle. Each cycle of therapy will be 3 weeks (21 days) in length. Control of local and metastatic disease can be achieved by radiation, surgery, local ablative techniques, or a combination of all three methods.

All patients will then receive 24 weeks of VINO-CPO maintenance. Each cycle of maintenance will be 28 days in length.

Patients will be asked to complete a patient medication diary for oral cyclophosphamide during Maintenance therapy.