Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma

Overview

  • Study type

    Interventional
  • Study phase

    III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 16-008368
    NCT ID: NCT02567435
    Sponsor Protocol Number: ARST1431

About this study

This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride) works compared to combination chemotherapy plus temsirolimus in treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as muscle), and has an intermediate chance of coming back after treatment (intermediate risk). Drugs used in chemotherapy 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. Combination chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether combination chemotherapy or combination chemotherapy plus temsirolimus is more effective in treating patients with intermediate-risk rhabdomyosarcoma.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Feasibility Phase: Patients must be < 21 years of age at the time of enrollment
  • Efficacy Phase: Patients must be < 40 years of age at the time of enrollment
  • 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, as below
  • RMS types included under embryonal rhabdomyosarcoma (ERMS) include those classified in the 1995 International Classification of Rhabdomyosarcoma (ICR) as ERMS (classic, spindle cell, and botryoid variants), which are reclassified in the 2013 World Health Organization (WHO) classification as ERMS (classic, dense and botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and the newly recognized sclerosing RMS variant); classification of alveolar rhabdomyosarcoma (ARMS) in the 2013 WHO classification is the same as in the ICR and includes classic and solid variants
    • ERMS
      • Stage 1, group III (non-orbit)
      • Stage 3, group I/II
      • Stage 2/3, group III
      • Stage 4, group IV, < 10 years old
    • ARMS:
      • Stages 1-3, groups I-III
  • Specimen Submission: Patients must have sufficient tissue available for the required biology studies
  • Lansky performance status score ≥ 50 for patients ≤ 16 years of age; Karnofsky performance status score ≥ 50 for patients > 16 years of age
  • Peripheral absolute neutrophil count (ANC) ≥ 750/uL
  • Platelet count ≥ 75,000/uL
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
    • 1 month to < 6 months old: 0.4 mg/dl (male), 0.4 mg/dl (female)
    • 6 months to < 1 year old: 0.5 mg/dl (male), 0.5 mg/dl (female)
    • 1 to < 2 years old: 0.6 mg/dl (male), 0.6 mg/dl (female)
    • 2 to < 6 years old: 0.8 mg/dl (male), 0.8 mg/dl (female)
    • 6 to < 10 years old: 1 mg/dl (male), 1 mg/dl (female)
    • 10 to < 13 years old: 1.2 mg/dl (male), 1.2 mg/dl (female)
    • 13 to < 16 years old: 1.5 mg/dl (male), 1.4 mg/dl (female)
    • ≥ 16 years old: 1.7 mg/dl (male), 1.4 mg/dl (female)
    • Patients with an elevated serum creatinine due to obstructive hydronephrosis secondary to tumor are still eligible; however, patients with urinary tract obstruction by tumor must have unimpeded urinary flow established via diversion (ie. percutaneous nephrostomies or ureteric stents) of the urinary tract
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
  • All patients and/or their parents or legal guardians must sign a written informed consent
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
  • FOXO1 Fusion Status:
    • All patients will undergo institutional pathology review and FOXO1 fusion determination; FOXO1 status results must be available by week 3 (day 21) of therapy; patients will be eligible to remain on protocol therapy based upon stage, group, and age:
      • FOXO1 fusion negative:
        • Stage 1, group III (non-orbit)
        • Stage 3, group I/II
        • Stage 2/3, group III
        • Stage 4, group IV, < 10 years old
      • FOXO1 fusion positive:
        • Stage 1-3, group I-III Note: FOXO1 fusion status must be performed at local institutions
  • Patients with institutional histologic classification of ARMS but FOXO1 fusion negative with the following stage and group can remain on study but will receive VAC/VA therapy on Regimen C instead of the previously assigned treatment regimen; patient consent is required to transfer to Regimen C
    • Stage 1, group I/II
    • Stage 1, group III (orbit)
    • Stage 2, group I/II

Exclusion Criteria:

  • Patients who have previously received TORI, another mTOR inhibitor, or any other investigational agent
  • Patients who have received any chemotherapy (excluding steroids) and/or radiation therapy prior to this enrollment
  • Patients with uncontrolled hyperglycemia
  • Patients with uncontrolled hyperlipidemia
  • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for at least 3 months after treatment is completed if sexually active with reproductive potential
  • Female patients who are pregnant are not eligible; Note: a pregnancy test is required for female patients of childbearing potential prior to study entry
  • Lactating females who plan to breastfeed their infants are not eligible

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Carola A Arndt, M.D.

Open for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

855-776-0015

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

Additional contact information

Cancer-related trials contact form

Phone: 855-776-0015 (toll-free)

International patient clinical studies questions