A Study to Investigate Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Systemic Lupus Erythematosus

NCT07630714 | Not Yet Recruiting Phase 2 FDA Drug FDA Device

• 3 other identifiers: D3465C000082025-524578-41-00EMA/PE/0000246211
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to characterize the pharmacokinetics (PK), pharmacodynamics (PD), and safety of subcutaneous (SC) anifrolumab in pediatric participants with moderate to severe systemic lupus erythematosus (SLE) while on background standard of care (SoC) therapy.

Conditions

Systemic Lupus Erythematosus

Keywords

Human immunoglobulin (Ig) G1κ monoclonal antibody (mAb) directed against subunit 1 of the type I interferon (IFN) receptor; Accessorized pre-filled syringe; Pharmacokinetics; Pharmacodynamics

Outcome Measures

Primary Outcomes (5)

• Maximum observed serum (peak) concentration (Cmax)

  To characterize PK (Cmax) of anifrolumab in pediatric participants with SLE following SC administration.

  Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

• Area under the serum concentration-time curve (AUC)

  To characterize PK (AUC) of anifrolumab in pediatric participants with SLE following SC administration.

  Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

• Time to maximum plasma concentration (tmax)

  To characterize PK (tmax) of anifrolumab in pediatric participants with SLE following SC administration.

  Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

• Apparent total body clearance of drug from plasma (CL/F)

  To characterize PK (CL/F) of anifrolumab in pediatric participants with SLE following SC administration.

  Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

• Trough drug concentration at steady state (Ctrough,ss)

  To characterize PK (Ctrough,ss) of anifrolumab in pediatric participants with SLE following SC administration.

  At Week 12

Secondary Outcomes (6)

• Suppression of type I IFN 21-gene signature

  At Week 12 and 52

• Change from baseline in anti-double-stranded deoxyribonucleic acid (dsDNA) antibodies

  At Week 12 and 52

• Change from baseline in complement component 3 (C3) levels

  At Week 12 and 52

• Change from baseline in complement component 4 (C4) levels

  At Week 12 and 52

• Change from baseline in total hemolytic complement (CH50) levels

  At Week 12 and 52

Other Outcomes (1)

• Number with participants with adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)

  Up to follow-up visit (12 weeks post last dose of study intervention) (approximately 64 weeks)

Study Arms (2)

Cohort 1 (body weight > 40 kg)

EXPERIMENTAL

Participants with body weight \> 40 kg will receive anifrolumab as an injection in APFS.

Combination Product: Anifrolumab + APFS

Cohort 2 (body weight ≥ 15 to ≤ 40 kg)

EXPERIMENTAL

Participants with body weight ≥ 15 to ≤ 40 kg will receive anifrolumab as an injection in APFS.

Combination Product: Anifrolumab + APFS

Interventions

Anifrolumab + APFS COMBINATION_PRODUCT

Anifrolumab will be administered as a SC injection using an APFS.

Also known as: MEDI-546, SAPHNELO™

Cohort 1 (body weight > 40 kg); Cohort 2 (body weight ≥ 15 to ≤ 40 kg)

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of SLE.
• Must be receiving at least one of the following SoC regimens for ≥ 4 weeks: oral glucocorticoids (≤1.0 mg/kg/day or ≤ 40 mg/day prednisone equivalent), antimalarials (hydroxychloroquine, chloroquine, or quinacrine), or a single permitted immunosuppressant (azathioprine, mycophenolate mofetil/mycophenolic acid, methotrexate, mizoribine, or tacrolimus) within specified dose limits.
• Participant must have moderate to severe active SLE disease defined as Systemic lupus erythematosus disease activity index 2000 (SLEDAI-2K) ≥ 6 total points.
• Body weight ≥ 15 kg.
• Participants must agree to follow study specific contraception requirements as per local regulations.

You may not qualify if:

• Known diagnosis of an IFN mediated autoinflammatory interferonopathy.
• History of, or current diagnosis of, clinically significant non-SLE related vasculitides.
• Active, severe SLE-driven renal disease with significant proteinuria.
• Active severe or unstable neuropsychiatric SLE including but not limited to aseptic meningitis; cerebral vasculitis; myelopathy; demyelination syndromes (ascending, transverse, acute inflammatory demyelinating polyradiculopathy); acute confusional state; impaired level of consciousness; psychosis; acute stroke or stroke syndrome; cranial neuropathy; status epilepticus; cerebellar ataxia; and mononeuritis multiplex.
• In participants ≥ 11 years of age, a history or evidence of suicidal ideation (severity of 4 \[active: method and intent, but no plan\] or 5 \[active: method, intent, and plan\]) within the past 6 months; or any suicidal behavior within the past 12 months or recurrent suicidal behavior in the lifetime of the participant based on an assessment with the Columbia suicide severity rating scale (C-SSRS).
• History of, or current diagnosis of, catastrophic antiphospholipid syndrome (APS).
• History of recurrent or opportunistic infection requiring hospitalization and intravenous (IV) antibiotics.
• Known history of a primary immunodeficiency, splenectomy, or any underlying condition that predisposes the participant to infection, or a positive result for human immunodeficiency virus (HIV) infection.
• Active hepatitis B and C infection.
• Any active or recent herpes zoster (HZ) infection that has not completely resolved within 12 weeks prior to study entry or that emerges between screening and Day 1.
• Any history of severe or recurrent HZ, including non-cutaneous HZ, herpes encephalitis, ophthalmic herpes, or 2 or more prior HZ episodes.
• Any cytomegalovirus (CMV) or Epstein Barr virus (EBV) infection that has not completely resolved.
• History of cancer.
• Prior receipt of anifrolumab.
• Prior treatment with directly acting cytotoxic B-cell depleting therapeutics.

Sponsors & Collaborators

• AstraZeneca: lead

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Mycobacterium Infections, Nontuberculous

Interventions

anifrolumab

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Mycobacterium Infections; Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2026
• First Posted: June 5, 2026
• Study Start (Estimated): July 28, 2026
• Primary Completion (Estimated): September 3, 2030
• Study Completion (Estimated): September 5, 2031
• Last Updated: June 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• 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 rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• 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. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.