Post-authorization safety study to evaluate the risks of myelodysplastic syndrome/acute myeloid leukemia and second primary malignancies in adult patients with platinum-sensitive, relapsed, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer receiving maintenance treatment with ZEJULA® (niraparib) (213705 - Zejula PASS 3000-04-001)

Study type

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

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This post-authorization safety study (PASS) will be conducted as a prospective, non-interventional, single-arm study. It will also utilize a retrospective control arm as an exploratory/descriptive comparison with the results collected prospectively.

Study drug and medical condition

• Name of medicine:
  ZEJULA
• Name of medicine, other:
  Niraparib
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XK02) niraparib

  niraparib
• Medical condition to be studied:
  Ovarian cancer

Population studied

• Age groups:
  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  800

Study design details

• Main study objective:
  The objective of this PASS is to determine the risk of developing MDS/AML and SPMs in patients administered niraparib in the routine clinical setting.
• Outcomes:
  Primary Objective: To estimate the incidence rate of MDS/AML among a cohort of adult patients with platinum-sensitive, relapsed, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer treated with niraparib who are in a complete or partial response to platinum-based chemotherapy.; Secondary Objective: To estimate the incidence rate of SPMs in the same cohort.
• Data analysis plan:
  The analysis population for all analyses is the safety population: all patients who receive any amount of niraparib (at least 1 dose). Analyses will be performed using SAS statistical software and include summary statistics (number and percentage for categorical variables and the number of patients, mean, standard deviation, median, minimum, and maximum for continuous variables). Analyses will be descriptive, no hypothesis will be tested. Distributions of patient and tumor characteristics will be summarized. Incidence rates of MDS/AML and other SPM and their respective 95% CIs per 100 person-time units will be estimated.The incidence of MDS/AML and SPM will be summarized across various potential risk factors for MDS/AML and SPM, such as: age, gender, chemo- and radiotherapy received, platelet transfusions, renal and/or hepatic insufficiency, CBC abnormalities, family history of MDS/AML, presence of autoimmune disorders, use of alcohol and/or tobacco and use of other PARP inhibitors.