Trabectedin First Line Therapy In Unfit Sarcoma Study
Condition: Metastatic and Locally Advanced Soft Tissue Tumor Patients Unfit to Receive; Standard Chemotherapy
MeSH terms: Soft Tissue Neoplasms
Study type: Interventional
Phase: Phase 2

Summary: 
      Phase II, non-randomized, two-stage study according to Bryant & Day The study enroll patients
      with Metastatic and locally advanced soft tissue sarcoma unfit to receive standard
      chemotherapy (doxorubicin/epirubicin and/or ifosfamide)
    

Detailed description: 
      Soft tissue sarcomas are a group of rare and aggressive disease, comprising more than a
      hundred different histological subtypes and mainly originating from the embryonic mesoderm.
      Today, they represent less than 1% of all adult cancers, with an incidence of 3/100000/year
      and a median age at diagnosis of 65 years. Despite the progress done in the last decade,
      approximately 50% of STS patients still develop distant metastases within 3 years from the
      diagnosis and die from their disease. Doxorubicin (or epirubicin) and ifosfamide have been
      proved to be active in the treatment of STS and they are widely used, alone or in
      combination, as a first line therapy for locally advanced and metastatic patients. However,
      the response rate to the combination regimen in non-pretreated patients does not exceed
      30-40%, and large randomized clinical trials failed to demonstrate any advantage in survival
      for the combination compared to single-agent treatment. Trabectedin (Yondelis®) is a
      marine-derived anticancer agent that has been approved in the European Union as a single
      agent for the treatment of STS patients after failure of standard chemotherapy (doxorubicin
      and/or ifosfamide) or for those unsuited to receive these agents. Even if the response rate
      in soft tissue sarcoma does not exceed 10%, trabectedin can provide a significant clinical
      benefit, by arresting disease progression in almost 50% of treated patients, with a
      progression-free survival rate of 20% at 6 months. Trabectedin was found to be particularly
      active in the treatment of myxoid liposarcoma and uterine leiomyosarcoma, for which better
      results have been obtained in terms of response rate and survival, suggesting an histotype
      driven activity. The toxicity profile of trabectedin given as second line therapy has been
      widely assessed in clinical studies and was largely manageable, with the majority of adverse
      events being grade 1 or 2 toxicities, generally reversible, dose or time dependent and
      noncumulative. The good tolerability profile observed in the trials seems to be confirmed
      also in everyday clinical practice. Conversely, few data are available at the moment about
      tolerability profile for those patients treated with trabectedin as first line because of
      medical conditions contraindicating the use of standard agents. The aim of this phase II
      study is to assess and describe trabectedin toxicity profile in this subset of negatively
      selected advanced inoperable STS patients.
    

Intervention name(s): Trabectedin
Intervention type(s): Drug
Intervention description(s): Each patients will receive intravenous trabectedin (1.5 mg/mq-1.3 mg/mq) over 24 hours every 3 weeks
Other intervention name(s): Yondelis
Intervention MeSH terms: Trabectedin
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Metastatic; Locally advanced; Soft tissue tumors