UK – A DISEASE REGISTRY STUDY TO PROSPECTIVELY OBSERVE TREATMENT PATTERNS AND OUTCOMES IN PATIENTS WITH HER2-POSITIVE UNRESECTABLE LOCALLY ADVANCED OR METASTATIC BREAST CANCER (ESTHER)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Disease epidemiology

  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Study drug and medical condition

• Name of medicine:
  Herceptin

  Kadcyla

  Perjeta
• Study drug International non-proprietary name (INN) or common name:
  PERTUZUMAB

  TRASTUZUMAB

  TRASTUZUMAB EMTANSINE
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XC03) trastuzumab

  (L01XC13) pertuzumab

  (L01XC14) trastuzumab emtansine
• Medical condition to be studied:
  Breast cancer
• Additional medical condition(s):
  HER2-Positive Unresectable Locally Advanced or Metastatic Breast Cancer

Population studied

• 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)
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  In patients with unresectable locally advanced (LA) or metastatic breast cancer (mBC), to observe the different anti-cancer treatment regimens and their sequencing throughout the course of the disease and to describe clinical outcome for each anti-cancer treatment regimen measured as progression-free survival (PFS).
• Setting:
  All eligible subjects were invited to participate in the study and enrolled sequentially. No other pre-selection criteria were applied.; Subjects met the following inclusion criteria for study entry:; - Males or females; - Initially diagnosed with HER2-positive unresectable LA/mBC no more than 6 months prior to enrolment, although they could have received anti-cancer treatment during that time; - Age ≥18 years; - Able and willing to provide written informed consent and to comply with the study protocol; No exclusion criteria were applied.
• Outcomes:
  In patients with unresectable LA/mBC, to observe the different anti-cancer treatment regimens and their sequencing throughout the course of the disease and to describe clinical outcome for each anti-cancer treatment regimen measured as progression-free survival (PFS). To observe safety profiles of different anti-cancer treatment regimens through reporting of serious adverse events (SAEs), specific adverse events relevant to HER2-targeted therapies and AEs leading to discontinuation or dose modification of an anti-cancer therapy, to describe incidence of and reasons for anti-cancer treatment modifications and treatment of populations of special interest.
• Data analysis plan:
  All enrolled patients who received at least one dose of an anti-cancer medication for HER2-positive unresectable LA/mBC will be included in the full analysis set, which will be the primary analysis population for safety and efficacy parameters. Other analysis populations may be defined based on more restrictive criteria. The analysis of the present study will be exploratory and primarily make use of descriptive statistical methods. In addition, exploratory statistical testing and modelling will be used to highlight interesting aspects of the data. Any test performed will be two-sided and carried out with a 5% α error rate without correction for multiplicity. A descriptive analysis of safety will be performed. The main safety parameter is the incidence of SAEs. The proportion of patients experiencing at least one event within each line of treatment will be estimated with 95% Clopper–Pearson CIs.

Documents

Study report

ML29659-Clinical_Study_Report_Synopsis_Redacted.pdf