Study type

Study topic

Human medicinal product

Study topic, other

Real World Safety and Effectiveness

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

ELREXFIO

Name of medicine, other

Elranatamab

Medical condition to be studied

Plasma cell myeloma refractory
Population studied

Short description of the study population

Male or female patients, age ≥18 years, with RRMM who are newly treated with elranatamab according to the local Health Authority approved product label (routine-care).

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

198
Study design details

Study design

This prospective, international, longitudinal cohort study will follow patients for up to 3 years or until withdrawal, physician discretion (i.e., patient health), loss to follow up, death, or study termination. Effectiveness outcomes and adverse events will be evaluated descriptively.

Main study objective

To evaluate the effectiveness of elranatamab through the collection and analysis of the following clinical outcomes (defined according to the IMWG consensus criteria for response and minimal residual disease assessment in MM): Overall response rate (ORR), Time to response (TTR), Duration of response (DOR), Progression free survival (PFS), Overall survival (OS), Time to next treatment (TTNT).

Setting

Patients will be recruited from primary care centers, hematology/oncology clinics, and academic treatment centers to ensure broad physician and patient representation. Recruitment of sites will begin with invitations to hematologists/oncologists who are most likely to treat patients with RRMM and will continue until the target number of patients has been met or 2 years after the first patient is enrolled. Approximately 198 patients will be recruited from primary care centers, hematology/oncology clinics, and academic centers from Germany, the United Kingdom, Brazil, Italy and Spain. Study will aim to enroll patients with a diverse distribution of characteristics (e.g., race, ethnicity, sex) that is representative of the real-world patient population being treated with elranatamab in clinical practice. Thus, both urban/university hospitals and rural community centers are included in this study.

Outcomes

Effectiveness outcomes include ORR, TTR, DOR, PFS, and OS according to the IMWG consensus criteria for response in MM. TTNT will be defined as the time from elranatamab initiation to next treatment. Safety event will be collected, reported and summarized by the enrolling physician or other treatment team member for up to 90 days after the last dose of elranatamab.

Data analysis plan

The characteristics captured during baseline and follow up will be summarized using descriptive statistics. Frequencies and percentages will be used for categorical variables and mean (standard deviation STD) and median (interquartile range IQR) will be used for continuous variables. For the effectiveness outcomes of interest, ORR will be summarized using frequencies and percentages and time-to-event outcomes (DOR, TTR, PFS, OS, TTNT) will be evaluated using Kaplan-Meier (KM) methods. KM curves will be illustrated and the median survival and corresponding 95% confidence interval (95% CI) will be computed. To evaluate the safety of elranatamab, AEs and SAEs will be collected on a structured data collection tool Adverse Event Monitoring (AEM) form and will be characterized by type, grade, timing, seriousness, and relationship to elranatamab. Crude cumulative incidence will be calculated as appropriate.