OPTIMIS - Outcomes of HCC patients treated with TACE followed or not followed by sorafenib and the influence of timing to initiate sorafenib

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary and secondary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  Nexavar
• Anatomical Therapeutic Chemical (ATC) code:
  100000096735

  sorafenib
• Medical condition to be studied:
  Hepatocellular carcinoma

Population studied

• Short description of the study population:
  Patient with a diagnosis of unresectable Hepatocellular carcinoma (HCC). Patients having following criteria were included:;  Patients with histologically/cytologically documented or radiographically diagnosed HCC. Radiographic diagnosis needs typical findings of HCC by radiographic method i.e. on multidimensional dynamic CT, CT hepatic arteriography (CTHA)/CT arterial portography (CTAP) or MRI.;  Patients with BCLC stage B or higher.;  Patients in whom a decision to treat with TACE has been made at time of study enrollment. Patients that have received one TACE in the past also can be enrolled, if the TACE was done at the same site and all required data about such previous TACEs are available. TACE includes both conventional TACE with lipidiol (or similar agents) and chemotherapeutic agent(s) and TACE with DC Beads® excluding TAE without chemotherapeutic agent.;  Patients with unresectable HCC (incurable with curative treatments including resection or ablation or not eligible for resection or local ablation);  Patients must have signed an informed consent form;  Patients must have a life expectancy of at least 8 weeks
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Hepatic impaired
• Estimated number of subjects:
  1670

Study design details

• Main study objective:
  The primary objective of this study is the comparison of two cohorts of hepatocellular carcinoma patients regarding overall survival (OS) from time of TACE non-eligibility. The two cohorts of special interest are defined based on the investigators’ treatment decisions (i.e. patients with early start
• Outcomes:
  Overall survival, defined as time (in days) from time of TACE non-eligibility to death due to any cause. Patients lost to follow-up or alive at the end of the study will be censored at the last date known to be alive. 1) Overall survival from initial TACE2) Progression-free survival from initial TACE3) Time to progression from initial TACE4) Tumor response according to mRECIST criteria5) Duration of treatment6) Number of patients with TEAEs (treatment emergent adverse events)For more secondary outcome measures please visit https://clinicaltrials.gov/ct2/show/NCT01933945
• Data analysis plan:
  In general, statistical analyses will be of explorative and descriptive nature. Analyses will be performed for the total study population (overall analysis) and separately for the two patient cohorts of special interest, as appropriate. The primary efficacy endpoint is Overall Survival (OS). It is defined in this study as the time period from documented TACE non-eligibility to death due to any cause. For the two cohorts of special interest, Kaplan-Meier (KM) estimates for OS will be displayed. Furthermore, these two cohorts will be compared regarding overall survival using a Cox proportional hazards model. Where applicable, the propensity score approach will be applied in order to compare the two cohorts.

Documents

Study results

16560_EU PAS_Abstract_2018-10-10

Study report

16560 OPTIMIS_Report Addendum_v1.0_20181015_Redacted

16560 OPTIMIS_Report_v1.0_20180529_Redacted

Study, other information

16560 OPTIMIS_Report_v1.0_20180529_Redacted.pdf