An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric Participants

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified April 2026 by AstraZeneca
Sponsor
AstraZeneca
Information Provided by (Responsible Party)
AstraZeneca
Clinicaltrials.gov Identifier
NCT05835310
Other Study ID Numbers:
D3461C00030
First Submitted
April 17, 2023
First Posted
April 27, 2023
Last Update Posted
May 5, 2026
Last Verified
April 2026

ClinicalTrials.gov processed this data on May 2026Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

This study aims to characterize the pharmacokinetics, pharmacodynamics, efficacy, and safety of anifrolumab solution for infusion compared with placebo solution for infusion in pediatric participants with severe active systemic lupus erythematosus who are on background standard of care therapy.

The study duration for a participant will be approximately 116 weeks, which includes:

* Screening period of up to 30 days.

* Part A consists of a four-week, double-blind, placebo-controlled, randomised, pharmacokinetic period.

* Part B is a double-blind, placebo-controlled, randomised, safety/efficacy period lasting 48 weeks (for rollover participants from Part A) or 52 weeks (for de novo participants).

* Part C is a 52-week open-label extension period.

* Part D is a safety follow-up period. One safety visit at 12 weeks post last dose.

Condition or DiseaseIntervention/Treatment
Systemic Lupus Erythematosus
Biological: AnifrolumabDrug: Placebo

Study Design

Study TypeInterventional
Actual Enrollment100 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingQuadruple
Primary PurposeTreatment
Official TitleA Phase III, Randomized, Double-blind, Parallel-group, Placebo-controlled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of IV Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Moderate to Severe Active Systemic Lupus Erythematosus While on Background Standard of Care Therapy
Study Start DateMarch 13, 2024
Actual Primary Completion Date2yrs 4mos from now
Actual Study Completion Date3yrs 7mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
Anifrolumab
Randomized participants will receive anifrolumab via intravenous (IV) infusion every 4 weeks
Biological: Anifrolumab
Participants will receive anifrolumab via IV infusion.
Placebo
Randomized participants will receive matching placebo via IV infusion
Drug: Placebo
Participants will receive matching placebo via IV infusion

Outcome Measures

Primary Outcome Measures
  1. Part A- Anifrolumab serum concentration
    The serum concentration will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severely active SLE.
  2. Part A - Maximum observed serum (peak) drug concentration (Cmax)
    The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severely active SLE.
  3. Part A - Area under the serum concentration curve (AUC)
    The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severe active SLE.
  4. Part A - Minimum observed serum concentration (Cmin)
    The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severe active SLE.
  5. Part B - Number of participants who are British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) responders (yes/no)
    BICLA response is defined as: * Reduction of all baseline British Isles Lupus Assessment Group BILAG-2004 A to B/C/D and B to C/D, and no BILAG-2004 worsening in other organ systems, as defined by &ge; 1 new BILAG-2004 A or &ge; 2 new BILAG- 2004 B. * No worsening from baseline in SLEDAI-2K, defined as an increase from baseline of \&gt; 0 points. * No worsening from baseline in participant&#039;s lupus disease activity, defined by an increase &ge; 0.30 points on a PGA 3-point visual analogue scale (VAS).
Secondary Outcome Measures
  1. Part B - Number of participants who are Systemic Lupus Erythematosus Responder Index of ≥ 4 SRI(4) responders (yes/no)
    SRI-4 response is defined as: * &ge; 4-point reduction from baseline in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score. * No new organ systems affected as defined by &ge; 1 new BILAG-2004 A or &ge; 2 new BILAG-2004 B items compared to baseline. * No worsening from baseline in participant&#039;s lupus disease activity, defined by an increase &ge; 0.30 points on a PGA 3-point VAS.
  2. Part B - Time to first flare
    Time to first flare, where flare is defined as either &ge; 1 new BILAG-2004 A, or &ge; 2 new BILAG-2004 B items compared with the previous visit.
  3. Part B - Anifrolumab serum concentration
    The PK of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  4. Part - B Change from baseline through Week 52 in antidrug antibody (ADA)
    The immunogenicity of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  5. Part - B Change from baseline in anti-double stranded deoxyribonucleic acid antibodies
    The PD of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  6. Part - B Change from baseline in total hemolytic complement (CH50)
    The PD of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  7. Part - B Change from baseline in complement component (C3)
    The PD of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  8. Part - B Change from baseline in complement component (C4)
    The PD of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
  9. Number of participants who are Pediatric Rheumatology International Trials Organization/American College of Rheumatology (PRINTO/ACR) childhood-onset systemic lupus erythematosus (cSLE) responders (yes/no)
    PRINTO/ACR cSLE responders are defined as participants with at least 50% improvement from baseline in any 2 of 5 core set outcome measures and no more than one of the remaining worsening more than 30%, where the core set measures are: * ParentGA 21-circle VAS * PGA 3-point VAS * SLEDAI-2K * PedsQL Generic Core (Physical Functioning Domain) * Proteinuria
  10. Part B - The mean percentage reduction from Baseline through Week 52 in oral corticosteroid(s) (OCS) background dose
    The efficacy of anifrolumab vs placebo on OCS background dose in pediatric participants with moderate to severe active SLE will be characterized.
  11. Part B - Change from baseline through Week 52 in type I interferon (IFN) 21-gene signature
    Type 1 IFN 21 gene signatures in pediatric patients with moderate to active SLE will be characterized.

Eligibility Criteria

Ages Eligible for Study(Child)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
Participant's parent/caregiver/legally authorized representative and participant (if required per local country regulation) capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Informed assent is to be provided by the participant per local country regulation.
Diagnosis of SLE according to the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) criteria for at least 3 months prior to signing the ICF.
Participant should meet all of following tuberculosis (TB) criteria: A. No signs or symptoms of active TB B. No medical history or past physical examinations suggestive of active TB C. No recent contact with a person with active TB or if there has been such contact, referral to a TB specialist for evaluation and initiation of treatment for latent TB, if warranted, prior to the first administration of study intervention in accordance with local SoC D. No history of latent TB without documented completion of treatment prior to initial screening visit
Female participants of childbearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test at randomization.
Female participants of childbearing and male participants must adhere to the contraception methods.
Exclusion Criteria
Known diagnosis of an IFN-mediated autoinflammatory interferonopathy.
History of, or current diagnosis of, clinically significant non-SLE-related vasculitides.
In participants aged 11 years and above: history or evidence of suicidal ideation.
History of any non-SLE disease that has required treatment with oral or parenteral corticosteroids for more than a total of 2 weeks within the last 24 weeks prior to signing the ICF.
Any positive result on screening for human immunodeficiency virus.
Active hepatitis B surface antigen OR hepatitis B core antibody (HBcAb), hepatitis C virus (HCV) antibody and detectable HCV ribonucleic acid (RNA) or any active or recent case of Herpes Zoster infection.
Any clinical cytomegalovirus or Epstein-Barr virus infection that has not completely resolved within 12 weeks prior to signing the ICF.
History of severe COVID-19 infection requiring hospitalization, intensive care unit care, or assisted ventilation or any prior COVID-19 infection with unresolved sequelae. Any mild/asymptomatic COVID-19 infection (laboratory confirmed or suspected based on clinical symptoms).
Prior use of anifrolumab.
Prior treatment with directly acting cytotoxic B-cell depleting therapeutics (eg, rituximab) \< 26 weeks prior to ICF signature.
Blood transfusion or receipt of blood products except albumin within 4 weeks prior to signing the ICF.

Contacts and Locations

Sponsors and CollaboratorsAstraZeneca
Locations
Research Site | Phoenix Arizona, United States, 85016Research Site | Los Angeles California, United States, 90027Research Site | Washington D.C. District of Columbia, United States, 20010Research Site | Chicago Illinois, United States, 60611Research Site | Chicago Illinois, United States, 60637Research Site | New Orleans Louisiana, United States, 70118Research Site | Bethesda Maryland, United States, 20889Research Site | Saint Paul Minnesota, United States, 55125Research Site | New Hyde Park New York, United States, 11042Research Site | New York New York, United States, 10032Research Site | The Bronx New York, United States, 10467Research Site | Valhalla New York, United States, 10595Research Site | Durham North Carolina, United States, 27710Research Site | Cincinnati Ohio, United States, 45229Research Site | Cleveland Ohio, United States, 44109Research Site | Columbus Ohio, United States, 43203Research Site | Portland Oregon, United States, 97227Research Site | Philadelphia Pennsylvania, United States, 19104Research Site | Greenville South Carolina, United States, 29605Research Site | El Paso Texas, United States, 79902Research Site | Houston Texas, United States, 77030Research Site | Salt Lake City Utah, United States, 84108Research Site | Buenos Aires , Argentina, C1270Research Site | Córdoba , Argentina, 5000Research Site | Rosario , Argentina, S2000PBJResearch Site | San Miguel de Tucumán , Argentina, T4000AXLResearch Site | Porto Alegre , Brazil, 90035-903Research Site | Ribeirão Preto , Brazil, 14048-900Research Site | São Paulo , Brazil, 04024-002Research Site | São Paulo , Brazil, 05403 000Research Site | Calgary British Columbia, Canada, T2N 4N1Research Site | Vancouver British Columbia, Canada, V6H 3N1Research Site | Toronto Ontario, Canada, M5G 1X8Research Site | Beijing , China, 100020Research Site | Beijing , China, 100032Research Site | Beijing , China, 100730Research Site | Changchun , China, 130021Research Site | Changsha , China, 410007Research Site | Nanjing , China, 210008Research Site | Shanghai , China, 201102Research Site | Suzhou , China, 215002Research Site | Wenzhou , China, 325027Research Site | Zhengzhou , China, 450018Research Site | Barranquilla , Colombia, 01800Research Site | Medellín , Colombia, 050034Research Site | Bordeaux , France, 33076Research Site | Bron , France, 69677Research Site | Le Kremlin-Bicêtre , France, 94275Research Site | Lille , France, 59037Research Site | Toulouse , France, 31300Research Site | Berlin , Germany, D-13353Research Site | Freiburg im Breisgau , Germany, 79106Research Site | Sankt Augustin , Germany, 53757Research Site | Genova , Italy, 16148Research Site | Milan , Italy, 20122Research Site | Milan , Italy, 20122Research Site | Padova , Italy, 35128Research Site | Roma , Italy, 00165Research Site | Bunkyō City , Japan, 113-8519Research Site | Bunkyō City , Japan, 113-8603Research Site | Chiba , Japan, 266-0007Research Site | Fuchu-shi , Japan, 183-8561Research Site | Kawasaki-shi , Japan, 216-8511Research Site | Kobe , Japan, 650-0047Research Site | Obu-shi , Japan, 474-8710Research Site | Shinjuku-ku , Japan, 162-8666Research Site | Yokohama , Japan, 232 8555Research Site | Yokohama , Japan, 236-0004Research Site | Atizapán de Zaragoza , Mexico, 52937Research Site | Guadalajara , Mexico, 44620Research Site | Mérida , Mexico, 97070Research Site | México , Mexico, 06720Research Site | Monterrey , Mexico, 64460Research Site | Lodź , Poland, 91-738Research Site | Warsaw , Poland, 02-637Research Site | Wroclaw , Poland, 52-114Research Site | Lisbon , Portugal, 1169-045Research Site | Lisbon , Portugal, 1649-035Research Site | Porto , Portugal, 4200-319Research Site | Cape Town , South Africa, 7700Research Site | Esplugues de Llobregat , Spain, 8950Research Site | Madrid , Spain, 28009Research Site | Madrid , Spain, 28034Research Site | Madrid , Spain, 28046Research Site | Málaga , Spain, 29011Research Site | Santiago de Compostela , Spain, 15706Research Site | Valencia , Spain, 46026Research Site | Ankara , Turkey (Türkiye), 06230Research Site | Istanbul , Turkey (Türkiye), 34098Research Site | Kayseri , Turkey (Türkiye), 38039Research Site | Umraniye , Turkey (Türkiye), 34760Research Site | Birmingham , United Kingdom, B4 6NHResearch Site | Bristol , United Kingdom, BS2 8BJResearch Site | Liverpool , United Kingdom, L12 2APResearch Site | London , United Kingdom, NW1 2PGResearch Site | London , United Kingdom, WC1N 3JHResearch Site | Manchester , United Kingdom, M13 9WLResearch Site | Southampton , United Kingdom, SO16 6YD