Saphnelo Use in Females of Child-bearing Potential (PRIMULA DUS)

A Non-Interventional Descriptive Multi-Country Study of Saphnelo™ (Anifrolumab-fnia) Utilization in Females of Reproductive Potential

Systemic lupus erythematosus (SLE) is a chronic autoimmune disease characterized by substantial clinical burden-including organ damage, increased morbidity, and mortality-that often presents in young adulthood and disproportionately affects female patients. SAPHNELO™ (anifrolumab-fnia), a fully human IgG1 κ monoclonal antibody, is a novel therapeutic option approved for add-on treatment of moderate-to-severe SLE in the United States (US) on 30 July 2021 and in the European Union on 14 February 2022. To fulfill US Food and Drug Administration (FDA) post-marketing requirements for the evaluation of anifrolumab safety in pregnancy, additional evidence is needed to better understand the real-world drug utilization of anifrolumab in female patients of reproductive potential.

Study Overview

• Status: Not yet recruiting
• Conditions: Systemic Lupus Erythematosus
• Intervention / Treatment: Drug: Anifrolumab

Detailed Description

Research Questions:

1. What is the incidence and prevalence of anifrolumab use among females of reproductive potential in selected countries where anifrolumab is marketed?
2. What are the baseline characteristics and drug utilization patterns in females of reproductive potential who initiate treatment with anifrolumab in selected countries where anifrolumab is marketed?

Primary Objectives:

1a. To describe trends in the annual incidence of anifrolumab utilization among females of reproductive potential overall and stratified by age group and, separately, by SLE diagnosis status.

1b. To describe trends in the annual prevalence of anifrolumab utilization among females of reproductive potential overall and stratified by age group and, separately, by SLE diagnosis status.

Secondary Objectives:

2. To describe the demographic and clinical characteristics of females of reproductive potential who initiate anifrolumab use, at the point of treatment initiation.

3. To describe SLE treatment regimens at anifrolumab initiation among females of reproductive potential with an SLE diagnosis.

4. To describe anifrolumab utilization patterns among females of reproductive potential who initiate anifrolumab use.

5. To enumerate and describe pregnancies with anifrolumab exposure among females of reproductive potential who initiate anifrolumab use.

This is a non-interventional, retrospective, descriptive cohort study using secondary healthcare data from selected countries where anifrolumab is marketed (specifically, Denmark, France, Germany, and the US) to assess trends in, and patterns of, anifrolumab use among females of reproductive potential.

The study population for the primary objectives will be female patients of reproductive potential (defined as females aged 15 to 50 years old) who are observable during the patient identification period (country-specific; market launch date through the end of available data). The study population for the secondary objectives will be female patients of reproductive potential who newly initiated anifrolumab use during the patient identification period. Secondary Objective 3 is restricted to female patients of reproductive potential who initiate anifrolumab use and have a prior record of SLE diagnosis. Study populations will be identified separately within each country-specific secondary healthcare data source from Denmark, France, Germany, and the US.

Final study report delivery is planned for April 2032.

• Study Type: Observational
• Enrollment (Estimated): 26000000

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population for the primary objectives will be all female patients of reproductive potential (defined as females aged 15 to 50 years old) observed during the patient identification period. The study population for the secondary objectives will be female patients of reproductive potential who newly initiated anifrolumab use during the patient identification period. Secondary Objective 3 is restricted to a subset of the broader secondary objective population, and includes female patients of reproductive potential who initiate anifrolumab use and have a prior record of SLE diagnosis. Study populations will be identified separately within each country-specific secondary healthcare data source from Denmark, France, Germany, and the US.

Description

Patients will be included in the primary objective cohort for a given calendar year interval, if they satisfy the following inclusion and exclusion criteria within that year:

Inclusion Criteria:

• Observability (enrollment or activity) in the data source for at least one day during the patient identification period within that calendar year (set earliest qualifying date as "index date" for primary objectives)
• Female sex at index date
• Age ≥15 and ≤50 years at index date

Exclusion Criteria:

• (Primary Objective 1a only) Evidence of prior anifrolumab use in all available data prior to the index date through 1 day prior to the index date

Patients will be included in the secondary objective cohort if they satisfy the following inclusion and exclusion criteria:

Inclusion criteria

• Receipt of anifrolumab therapy during the patient identification period (identify earliest record of anifrolumab in this period and set as "index date" for secondary objectives).
• Female sex at index date.
• Age ≥15 and ≤50 years at index date.
• ≥180 days of continuous observability (e.g., enrollment or activity) in the dataset prior to and including the index date.
• (For Secondary Objective 3 only) SLE diagnosis, defined as having at least one unique health care encounter record with an International Classification of Diseases, 10th Revision (ICD-10) code of M32* (excluding M32.0: drug-induced SLE) within the 180-day baseline period prior to and including the index date.

Exclusion criteria

• Evidence of prior anifrolumab use in all available data prior the index date through 1 day prior to the index date.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Study population for primary objectives; Patients will be included in the primary objective cohort for a given calendar year interval, if they satisfy the following inclusion and exclusion criteria within that year: Inclusion criteria; Observability (enrollment or activity) in the data source for at least one day during the patient identification period within that calendar year (set earliest qualifying date as "index date" for primary objectives); Female sex at index date; Age ≥15 and ≤50 years at index date Exclusion criteria 1. (Primary Objective 1a only) Evidence of prior anifrolumab use in all available data prior to the index date through 1 day prior to the index date Note: Cohort selection criteria for the primary objectives will be assessed repeatedly within calendar year intervals during the patient identification period to enable evaluation of annual anifrolumab use incidence and prevalence outcomes.	Drug: Anifrolumab; Anifrolumab is a human monoclonal antibody that binds to subunit 1 of the type 1 interferon receptor, which was developed based on the evidence supporting the role of type 1 interferon pathway in SLE. Clinical trial evidence from TULIP 1 and TULIP 2 have showed that monthly intravenous administration of anifrolumab led to a higher percentage of patients with a response, assessed with the British Isles Lupus Assessment Group-based Composite Lupus Assessment, compared with patients receiving placebo. Moreover, the phase II MUSE study showed that administration of anifrolumab resulted in substantially reduce disease activity, as measured by the SLE Responder Index, compared to patients receiving placebo. Anifrolumab was approved by the FDA and EMA in July 2021 and February 2022, respectively, for the treatment of adult patients with moderate to severe SLE who are receiving standard therapy.; Other Names: Saphnelo
Study population for secondary objectives; Patients will be included in the secondary objective cohort if they satisfy the following inclusion and exclusion criteria: Inclusion criteria; Receipt of anifrolumab therapy during the patient identification period (identify earliest record of anifrolumab in this period and set as "index date" for secondary objectives); Female sex at index date; Age ≥15 and ≤50 years at index date; ≥180 days of continuous observability (e.g., enrollment or activity) in the dataset prior to and including the index date.; (For Secondary Objective 3 only) SLE diagnosis, defined as having at least one unique health care encounter record with an International Classification of Diseases, 10th Revision (ICD-10) code of M32* (excluding M32.0: drug-induced SLE) within the 180-day baseline period prior to and including the index date Exclusion criteria 1. Evidence of prior anifrolumab use in all available data prior the index date through 1 day prior to the index date	Drug: Anifrolumab; Anifrolumab is a human monoclonal antibody that binds to subunit 1 of the type 1 interferon receptor, which was developed based on the evidence supporting the role of type 1 interferon pathway in SLE. Clinical trial evidence from TULIP 1 and TULIP 2 have showed that monthly intravenous administration of anifrolumab led to a higher percentage of patients with a response, assessed with the British Isles Lupus Assessment Group-based Composite Lupus Assessment, compared with patients receiving placebo. Moreover, the phase II MUSE study showed that administration of anifrolumab resulted in substantially reduce disease activity, as measured by the SLE Responder Index, compared to patients receiving placebo. Anifrolumab was approved by the FDA and EMA in July 2021 and February 2022, respectively, for the treatment of adult patients with moderate to severe SLE who are receiving standard therapy.; Other Names: Saphnelo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of anifrolumab use	Within each country, the incidence of anifrolumab will be assessed within each calendar year interval of the overall patient identification period. The follow-up period within each calendar year interval starts on the calendar year-specific index date and ends on the earliest of the following dates within that same year: (1) last date of observability (enrollment/activity), (2) death date, (3) 51st birthday, (4) end of the study period, (5) December 31st (end of calendar year), or (6) date of anifrolumab initiation (for objective 1a only). Incidence will be reported as the number of female patients of reproductive potential with incident anifrolumab use per (1) 1,000,000 females of reproductive potential (without prior anifrolumab use) in a given calendar year, with 95% CI and (2) per 1,000,000 person-years of follow-up among females of reproductive potential (without prior anifrolumab use) in a given calendar year, with 95% CI.	1 year
Prevalence of anifrolumab use	Within each country, the prevalence of anifrolumab use will be assessed within each calendar year interval of the follow-up period by calculating the number and percentage with prevalent anifrolumab use among all female patients of reproductive potential in a given calendar year (with 95% CI), as well as number of female patients of reproductive potential with prevalent anifrolumab use per 1,000,000 female patients of reproductive potential in a given calendar year (with 95% CI).	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Baseline patient characteristics	Patient demographics and clinical characteristics for the secondary objective cohort will be assessed using descriptive statistics over the appropriate baseline period. Categorical variables will be reported as counts with percentages, and continuous variables will be reported as mean (SD) and median (IQR). The frequency (and proportion) of missing responses will be reported for baseline patient characteristics.	At anifrolumab treatment initiation (index date)
Baseline SLE treatment characteristics	Baseline SLE treatment utilization characteristics will be assessed over the appropriate baseline period among the patients in the secondary objective cohort with a baseline SLE diagnosis. The number and percentage of patients who have a record for each unique SLE treatment during the baseline assessment period will be reported.	At anifrolumab treatment initiation (index date)
Anifrolumab treatment utilization patterns	Anifrolumab treatment use outcomes will be assessed in the secondary objective cohort from one day after the index date until the earliest of: last date of observability, death, 51st birthday, end of study period, or anifrolumab discontinuation (end of first episode of use). Duration of anifrolumab use (in days) will be estimated using the Kaplan-Meier method, summarized as (1) median time on treatment (with 95% CI) and (2) proportion of patients still on treatment at 84 days (with 95% CI). Additional outcome measures include total count of doses per patient (mean, SD, median, IQR, range), and proportion with anifrolumab discontinuation (defined as a gap of 90+ days between infusions, with discontinuation date as 28 days after last dose before discontinuation) during follow-up.	From anifrolumab treatment initiation (index date) up to 84 days post-initiation
Anifrolumab-exposed pregnancies	Pregnancies that occur during anifrolumab exposure will be identified and assessed in the secondary objective cohort from one day after the index date until the earliest of: last date of observability, death, 51st birthday, end of study period, or last date of anifrolumab exposure (set as 93 days after last dose in first episode of use). Reported outcome measures will include: (1) total count of anifrolumab-exposed pregnancy episodes (cohort-level), (2) counts with percentages of patients who initiated anifrolumab use with 0, 1, or ≥2 unique anifrolumab-exposed pregnancy episodes, (3) number (and percentage) of patients who were pregnant at time of anifrolumab initiation, (4) trimester of pregnancy at anifrolumab initiation (counts/percentages for 1st, 2nd, or 3rd trimester), and (5) duration of anifrolumab exposure (mean, SD, median, IQR, range) among anifrolumab-exposed pregnancy episodes.	From anifrolumab treatment initiation (index date) up to 93 days after date of last dose

Collaborators and Investigators

• Sponsor: AstraZeneca
• Collaborators: Aetion, Inc.
• Investigators: Study Director: Zachary Bouck, AstraZeneca

Study record dates

Study Major Dates

• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): March 31, 2032
• Study Completion (Estimated): March 31, 2032

Study Registration Dates

• First Submitted: January 12, 2026
• First Submitted That Met QC Criteria: February 20, 2026
• First Posted (Actual): February 25, 2026

Study Record Updates

• Last Update Posted (Actual): February 25, 2026
• Last Update Submitted That Met QC Criteria: February 20, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: pregnancy; Systemic Lupus Erythematosus; corticosteroids; immunosuppressants; chronic autoimmune disease; human monoclonal antibody (IgG1ƙ mAb); post Marketing Requirements (PMR) study
• Additional Relevant MeSH Terms: Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin and Connective Tissue Diseases; Lupus Erythematosus, Systemic; anifrolumab
• Other Study ID Numbers: D3461R00086

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No