Anifrolumab for Lupus

(BLOSSOM Trial)

Recruiting in Palo Alto (17 mi)

+80 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: AstraZeneca

Must not be taking: Cytotoxic B-cell depleters

Disqualifiers: HIV, Hepatitis B, Hepatitis C, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

A Study to evaluate the PK, PD, efficacy, and safety of Anifrolumab in children with moderate to severe active SLE

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you have used certain treatments like Anifrolumab or B-cell depleting therapies recently, you may not be eligible to participate.

What data supports the effectiveness of the drug Anifrolumab for lupus?

Anifrolumab has been shown to be effective in treating systemic lupus erythematosus (SLE), a condition where the immune system attacks the body's own tissues, and lupus nephritis, a kidney inflammation caused by lupus. It works by blocking a specific protein involved in the immune response, and has been approved in the USA for adults with moderate to severe SLE who are already on standard therapy.

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Is Anifrolumab safe for humans?

Anifrolumab has been studied for safety in people with systemic lupus erythematosus (SLE). In clinical trials, serious side effects were reported in 8-16% of patients taking Anifrolumab, compared to 16-19% of those taking a placebo. However, there was a higher incidence of herpes zoster (shingles) in patients taking Anifrolumab (up to 7%) compared to those on placebo (up to 2%).

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What makes the drug Anifrolumab unique for treating lupus?

Anifrolumab is unique because it is a monoclonal antibody that specifically targets and blocks the type I interferon receptor, which plays a key role in the development of systemic lupus erythematosus (SLE). This mechanism of action is different from other lupus treatments, and it has shown effectiveness in reducing disease activity and the need for oral corticosteroids in clinical trials.

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Eligibility Criteria

This trial is for children with moderate to severe active Systemic Lupus Erythematosus (SLE). Participants need consent from a parent or guardian, must meet specific SLE criteria, have no signs of active tuberculosis (TB), and girls able to have babies must not be pregnant and agree to use birth control. A negative COVID-19 test is also required.

Inclusion Criteria

I am a woman who can have children and my pregnancy test was negative.

I am committed to using birth control as required.

I have been diagnosed with lupus according to the 2019 EULAR/ACR criteria.

Exclusion Criteria

I have previously been treated with Anifrolumab.

I have active hepatitis B, hepatitis C, or a severe case of Herpes Zoster.

Any positive result on Screening for human immunodeficiency virus

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Part A - Pharmacokinetic Period

A single-blind, placebo-controlled, randomised pharmacokinetic period

4 weeks

Weekly visits for infusion and monitoring

Part B - Safety/Efficacy Period

Double-blind, placebo-controlled, randomised safety and efficacy period

48-52 weeks

Monthly visits for infusion and assessments

Part C - Open-label Extension

Participants may continue treatment in an open-label extension

52 weeks

Monthly visits for infusion and assessments

Part D - Safety Follow-up

Participants are monitored for safety 12 weeks post last dose

12 weeks

One safety visit

Participant Groups

The study tests the effectiveness and safety of Anifrolumab given through an IV compared to a placebo in treating pediatric SLE. It will assess how the drug moves through and affects the body, as well as its ability to improve symptoms.

Group I: Anifrolumab Experimental Treatment 1 Intervention

Randomized participants will receive a single dose of Anifrolumab via IV infusion every 4 weeks

Group II: Placebo Placebo Group 1 Intervention

Randomized participants will receive matching placebo via IV infusion

Anifrolumab is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Saphnelo for:

• Moderate to severe systemic lupus erythematosus (SLE)

🇪🇺 Approved in European Union as Saphnelo for:

• Moderate to severe systemic lupus erythematosus (SLE)

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Research Site Columbus, OH

Research Site Phoenix, AZ

Research Site Washington, United States

Research Site New Hyde Park, NY

More Trial Locations

Who Is Running the Clinical Trial?

AstraZeneca Lead Sponsor

References

1. England pubmed.ncbi.nlm.nih.gov

Phase II randomised trial of type I interferon inhibitor anifrolumab in patients with active lupus nephritis. [2022] To assess the efficacy and safety of the type I interferon receptor antibody, anifrolumab, in patients with active, biopsy-proven, Class III/IV lupus nephritis.

2. United States pubmed.ncbi.nlm.nih.gov

Long-Term Safety and Efficacy of Anifrolumab in Adults With Systemic Lupus Erythematosus: Results of a Phase II Open-Label Extension Study. [2021] To investigate long-term safety and tolerability of anifrolumab, a human monoclonal antibody to the type I interferon (IFN) receptor subunit 1, in patients with moderate-to-severe systemic lupus erythematosus (SLE).

3. England pubmed.ncbi.nlm.nih.gov

Anifrolumab in lupus nephritis: results from second-year extension of a randomised phase II trial. [2023] To characterise the safety and efficacy of anifrolumab in active lupus nephritis (LN) through year 2 of the phase II randomised, double-blind Treatment of Uncontrolled Lupus via the Interferon Pathway (TULIP)-LN trial (NCT02547922) of 2 anifrolumab dosing regimens versus placebo.

4. New Zealand pubmed.ncbi.nlm.nih.gov

Anifrolumab: First Approval. [2022] Anifrolumab (anifrolumab-fnia; Saphnelo&#8482;) is a monoclonal antibody antagonist of the type 1 interferon receptor (IFNAR). It is being developed by AstraZeneca (under license from Medarex, now Bristol-Myers Squibb) for the treatment of autoimmune disorders, including systemic lupus erythematosus (SLE) and lupus nephritis, the underlying pathogenesis of which involves type 1 interferon. In July 2021, intravenous anifrolumab was approved in the USA for the treatment of adult patients with moderate to severe SLE who are receiving standard therapy. Anifrolumab (intravenous or subcutaneous) continues to be assessed in clinical studies in SLE in various countries, and the intravenous formulation is under regulatory review in the EU and Japan. This article summarizes the milestones in the development of anifrolumab leading to this first approval for the treatment of moderate to severe SLE.

5. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of anifrolumab therapy in systemic lupus erythematosus in real-world clinical practice: LOOPS registry. [2023] To determine the safety and efficacy of anifrolumab in patients with systemic lupus erythematosus (SLE) classified based on the Lupus Low Disease Activity State (LLDAS) in real-world clinical practice.

6. England pubmed.ncbi.nlm.nih.gov

Anifrolumab, a monoclonal antibody to the type I interferon receptor subunit 1, for the treatment of systemic lupus erythematosus: an overview from clinical trials. [2021] Chronic activation of the type I interferon (IFN) pathway plays a critical role in systemic lupus erythematosus (SLE) pathogenesis. Anifrolumab is a human monoclonal antibody to the type I IFN receptor subunit 1, which blocks the action of type I IFNs. Two phase 3 studies (TULIP-1 and TULIP-2) and a phase 2b study (MUSE) provide substantial evidence for the efficacy and safety of anifrolumab for moderately to severely active SLE. In all three studies, monthly intravenous anifrolumab 300 mg was associated with treatment differences >16% compared with placebo at Week 52 in British Isles Lupus Assessment Group-based Composite Lupus Assessment response rates. The combined data across a range of other clinically significant endpoints (e.g. oral corticosteroid reduction, improved skin disease, flare reduction) further support the efficacy of anifrolumab for SLE treatment. The safety profile of anifrolumab was generally similar across all studies; serious adverse events occurred in 8-16% and 16-19% of patients receiving anifrolumab and placebo, respectively. Herpes zoster incidence was greater with anifrolumab (≤7%) vs placebo (≤2%). Evidence from these clinical trials suggests that in patients with active SLE, anifrolumab is superior to placebo in achieving composite endpoints of disease activity response and oral corticosteroid reduction.

7. United States pubmed.ncbi.nlm.nih.gov

A Randomized, Placebo-Controlled Phase III Extension Trial of the Long-Term Safety and Tolerability of Anifrolumab in Active Systemic Lupus Erythematosus. [2023] To explore long-term safety and tolerability of anifrolumab 300 mg compared with placebo in patients with systemic lupus erythematosus (SLE) who completed a Treatment of Uncontrolled Lupus via the Interferon Pathway (TULIP) trial and enrolled in the placebo-controlled 3-year long-term extension (LTE) study (ClinicalTrials.gov identifier: NCT02794285).

8. United States pubmed.ncbi.nlm.nih.gov

Interferon Inhibition for Lupus with Anifrolumab: Critical Appraisal of the Evidence Leading to FDA Approval. [2022] Furie R, Khamashta M, Merrill JT, Werth VP, Kalunian K, Brohawn P, et al. Anifrolumab, an anti-interferon-α receptor monoclonal antibody, in moderate-to-severe systemic lupus erythematosus. Arthritis Rheumatol 2017;69:376-86. Objective To assess the efficacy and safety of anifrolumab, a type I interferon (IFN) receptor antagonist, in a phase IIb, randomized, double-blind, placebo-controlled study of adults with moderate-to-severe systemic lupus erythematosus (SLE). Methods Patients (n = 305) were randomized to receive intravenous anifrolumab (300 mg or 1,000 mg) or placebo, in addition to standard therapy, every 4 weeks for 48 weeks. Randomization was stratified by SLE Disease Activity Index 2000 score (

9. England pubmed.ncbi.nlm.nih.gov

Anifrolumab effects on rash and arthritis: impact of the type I interferon gene signature in the phase IIb MUSE study in patients with systemic lupus erythematosus. [2022] This post hoc analysis compared anifrolumab 300 mg every 4 weeks with placebo on rash and arthritis measures with different stringency in patients with moderate to severe SLE (phase IIb; MUSE; NCT01438489). Subgroups were analysed by type I interferon gene signature (IFNGS test-high or test-low).