Prospective non-interventional post authorization safety study (PASS) of idelalisib in Germany

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)

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Chronic lymphocytic leukaemia

  Follicular lymphoma

Population studied

• Short description of the study population:
  The study population will include 300 adult patients (≥ 18 years) with Chronic lymphocytic leukaemia (CLL) or Follicular lymphoma (FL).; ; Inclusion Criteria CLL; 1) Diagnosis of chronic lymphocytic leukemia (CLL) and decision for treatment with idelalisib; 2) Indication approved for Zydelig in the European Union (EU) (refer to current Zydelig EU SmPC); 3) Understand and voluntarily sign an informed consent form; 4) Male or female ≥18 years of age at the time of signing the informed consent form; ; Exclusion Criteria CLL; 1) Patients with suspected systemic bacterial, fungal or viral infection; 2) Patients with history of another primary malignancy that is currently clinically significant or currently requires active intervention; 3) Pregnant or breast feeding women; 4) Concurrent participation in another therapeutic clinical trial; ; Inclusion / Exclusion Criteria FL (Cohort B); Inclusion Criteria FL; 1) Histopathologically confirmed diagnosis of Follicular Lymphoma and decision for treatment with idelalisib; 2) Indication approved for Zydelig in the European Union (EU) (refer to current Zydelig EU SmPC); 3) Understand and voluntarily sign an informed consent form; 4) Male or female ≥18 years of age at the time of signing the informed consent form; ; Exclusion Criteria FL; 1) Patients with suspected systemic bacterial, fungal or viral infection; 2) Patients with history of another primary malignancy that is currently clinically significant or currently requires active intervention; 3) Patients must not have received autologous stem cell transplant at least within 12 weeks prior to study treatment. If patients received autologous stem cell transplant more than 12 weeks ago, they must be fully recovered from the side effects of such treatment; 4) Pregnant or breast feeding women; 5) Concurrent participation in another therapeutic clinical trial
• 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:
  Other
• Special population of interest, other:
  Chronic lymphocytic leukaemia (CLL) or Follicular lymphoma (FL) patients
• Estimated number of subjects:
  179

Study design details

• Main study objective:
  The primary objectives of this study are to assess: Progression-Free Survival (PFS) (rate and time to progression) Overall Response Rate (ORR) Overall Survival (OS) (rate and survival duration)
• Outcomes:
  The primary objectives of this study are to assess: Progression-Free Survival (PFS) (rate and time to progression) Overall Response Rate (ORR) Overall Survival (OS) (rate and survival duration), Incidence of adverse drug reactions (ADRs), serious ADRs, fatal events (regardless of causality) and cause of death Incidence, risk factors, management & outcome of diarrhea/colitis, pneumonitis and liver enzyme elevation Type and incidence of Special Situation Reports Health resource utilization Patient Reported Outcome (health-related quality of life & health status)
• Data analysis plan:
  Summary statistics were presented by cohort (CLL or FL) and included: -nominal variables: frequencies and percentages. -ordinal variables: frequencies, percentages, median, minimum and maximum. -continuous variables: number (N) of observations, mean, standard deviation, 25th percentile, median, 75th percentile, minimum and maximum. For ADR events, in addition to frequencies and percentages, incidence rate in person-time were calculated by dividing number of new cases by the total number of person-time at risk to account for varying length of follow-up. Kaplan-Meier plots of progression-free survival, overall response rate, and overall survival were produced. The median duration of progression-free survival, overall response rate, and overall survival were determined. If not otherwise specified, p-values were presented as two-sided and the level of significance was set to 5% (two-sided). 95%-confidence intervals were provided, where applicable.