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NCT05835310PHASE3Recruiting

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

AstraZeneca

Start Date

3/14/2024

Completion Date

1/9/2030

Summary

A Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Efficacy, and Safety of Anifrolumab in Children with Moderate to Severe Active Systemic Lupus Erythematosus (SLE)

Detailed 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.

Eligibility Criteria

Age Range: 5 years to 17 years

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. * At Screening, participant must have moderate to severe active SLE disease, as adjudicated by the Central Adjudication Committee, defined as: (a) SLEDAI-2K activity of: (i) ≥ 6 points with at least 4 points (≥ 4 points) coming from the following clinical components ('Clinical' SLEDAI-2K score): arthritis, myositis, rash, alopecia, mucosal ulcers, pleurisy, pericarditis, or vasculitis and excluding points attributed to a fever, SLE headache, and organic brain syndrome (ii) Clinical SLEDAI score of ≥ 4 points verified at Day 1 (b) BILAG-2004 activity of: (i) ≥ 1 BILAG A score; or (ii) ≥ 2 BILAG B scores (c) PGA score ≥ 1.0 on a 0 to 3 VAS * 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.

Interventions

BIOLOGICAL

Anifrolumab

DRUG

Placebo

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Conditions

Systemic Lupus Erythematosus

Locations

Research Site

Phoenix, Arizona 85016

United States

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Los Angeles, California 90027

United States

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Washington D.C., District of Columbia 20010

United States

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Chicago, Illinois 60611

United States

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Chicago, Illinois 60637

United States

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New Orleans, Louisiana 70118

United States

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Bethesda, Maryland 20889

United States

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Saint Paul, Minnesota 55125

United States

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New Hyde Park, New York 11042

United States

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New York, New York 10032

United States

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The Bronx, New York 10467

United States

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Valhalla, New York 10595

United States

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Durham, North Carolina 27710

United States

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Cincinnati, Ohio 45229

United States

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Cleveland, Ohio 44109

United States

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Columbus, Ohio 43203

United States

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Portland, Oregon 97227

United States

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Philadelphia, Pennsylvania 19104

United States

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Greenville, South Carolina 29605

United States

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El Paso, Texas 79902

United States

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Houston, Texas 77030

United States

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Salt Lake City, Utah 84108

United States

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Buenos Aires, C1270

Argentina

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Córdoba, 5000

Argentina

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Rosario, S2000PBJ

Argentina

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San Miguel de Tucumán, T4000AXL

Argentina

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Belo Horizonte, 30130-100

Brazil

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Porto Alegre, 90035-903

Brazil

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Ribeirão Preto, 14048-900

Brazil

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São Paulo, 04024-002

Brazil

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São Paulo, 05403 000

Brazil

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Calgary, British Columbia T2N 4N1

Canada

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Vancouver, British Columbia V6H 3N1

Canada

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Toronto, Ontario M5G 1X8

Canada

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Beijing, 100020

China

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Beijing, 100032

China

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Beijing, 100730

China

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Changchun, 130021

China

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Changsha, 410007

China

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Nanjing, 210008

China

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Shanghai, 201102

China

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Suzhou, 215002

China

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Wenzhou, 325027

China

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Zhengzhou, 450018

China

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Barranquilla, 01800

Colombia

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Medellín, 050034

Colombia

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Bordeaux, 33076

France

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Bron, 69677

France

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Le Kremlin-Bicêtre, 94275

France

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Lille, 59037

France

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Toulouse, 31300

France

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Berlin, D-13353

Germany

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Freiburg im Breisgau, 79106

Germany

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Sankt Augustin, 53757

Germany

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Genova, 16148

Italy

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Milan, 20122

Italy

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Milan, 20122

Italy

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Padova, 35128

Italy

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Roma, 00165

Italy

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Bunkyō City, 113-8519

Japan

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Bunkyō City, 113-8603

Japan

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Chiba, 266-0007

Japan

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Fuchu-shi, 183-8561

Japan

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Kawasaki-shi, 216-8511

Japan

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Kobe, 650-0047

Japan

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Obu-shi, 474-8710

Japan

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Shinjuku-ku, 162-8666

Japan

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Yokohama, 232 8555

Japan

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Yokohama, 236-0004

Japan

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Atizapán de Zaragoza, 52937

Mexico

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Guadalajara, 44620

Mexico

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Mérida, 97070

Mexico

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México, 06720

Mexico

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Monterrey, 64460

Mexico

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Lodź, 91-738

Poland

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Warsaw, 02-637

Poland

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Wroclaw, 52-114

Poland

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Lisbon, 1169-045

Portugal

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Lisbon, 1649-035

Portugal

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Porto, 4200-319

Portugal

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Cape Town, 7700

South Africa

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Esplugues de Llobregat, 8950

Spain

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Madrid, 28009

Spain

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Madrid, 28034

Spain

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Madrid, 28046

Spain

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Málaga, 29011

Spain

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Santiago de Compostela, 15706

Spain

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Valencia, 46026

Spain

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Ankara, 06230

Turkey (Türkiye)

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Istanbul, 34098

Turkey (Türkiye)

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Kayseri, 38039

Turkey (Türkiye)

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Umraniye, 34760

Turkey (Türkiye)

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Birmingham, B4 6NH

United Kingdom

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Bristol, BS2 8BJ

United Kingdom

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Liverpool, L12 2AP

United Kingdom

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London, NW1 2PG

United Kingdom

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London, WC1N 3JH

United Kingdom

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Manchester, M13 9WL

United Kingdom

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Southampton, SO16 6YD

United Kingdom