Anifrolumab: Participants will receive anifrolumab via IV infusion.
Placebo: Participants will receive matching placebo via IV infusion
Study 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)
Eligibility
Sex
ALL
Min age
5 Years
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Part A- Anifrolumab serum concentration — Pre-dose Day 29 The serum concentration will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severely active SLE.
Part A - Maximum observed serum (peak) drug concentration (Cmax) — Up to Day 29 The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severely active SLE.
Part A - Area under the serum concentration curve (AUC) — Up to Day 29 The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severe active SLE.
Part A - Minimum observed serum concentration (Cmin) — Up to Day 29 The serum PK will be characterised and the dose of anifrolumab will be defined in pediatric participants with moderate to severe active SLE.
Part B - Number of participants who are British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) responders (yes/no) — At Week 52 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 ≥ 1 new BILAG-2004 A or ≥ 2 new BILAG- 2004 B.
* No worsening from baseline in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), defined as an increase from baseline of \> 0 points.
* No worsening from baseline in participant's lupus disease activity, defined by an increase ≥ 0.30 points on a Physician's Global Assessment (PGA) 3-point visual analogue scale (VAS).
Trial sites (103)
Facility
City
Region
Status
Research Site
Phoenix
Arizona
Recruiting
Research Site
Los Angeles
California
Recruiting
Research Site
Washington D.C.
District of Columbia
Recruiting
Research Site
Chicago
Illinois
Recruiting
Research Site
Chicago
Illinois
Recruiting
Research Site
New Orleans
Louisiana
Recruiting
Research Site
Bethesda
Maryland
Withdrawn
Research Site
Saint Paul
Minnesota
Recruiting
Research Site
New Hyde Park
New York
Recruiting
Research Site
New York
New York
Recruiting
Research Site
The Bronx
New York
Recruiting
Research Site
Valhalla
New York
Recruiting
Research Site
Durham
North Carolina
Recruiting
Research Site
Cincinnati
Ohio
Recruiting
Research Site
Cleveland
Ohio
Recruiting
Research Site
Columbus
Ohio
Recruiting
Research Site
Portland
Oregon
Recruiting
Research Site
Philadelphia
Pennsylvania
Recruiting
Research Site
Greenville
South Carolina
Withdrawn
Research Site
El Paso
Texas
Recruiting
Research Site
Houston
Texas
Recruiting
Research Site
Salt Lake City
Utah
Recruiting
Research Site
Buenos Aires
Argentina
Withdrawn
Research Site
Córdoba
Argentina
Recruiting
Research Site
Rosario
Argentina
Recruiting
Research Site
San Miguel de Tucumán
Argentina
Recruiting
Research Site
Belo Horizonte
Brazil
Not Yet Recruiting
Research Site
Belo Horizonte
Brazil
Not Yet Recruiting
Research Site
Porto Alegre
Brazil
Recruiting
Research Site
Ribeirão Preto
Brazil
Recruiting
Research Site
São Paulo
Brazil
Recruiting
Research Site
São Paulo
Brazil
Recruiting
Research Site
Calgary
British Columbia
Not Yet Recruiting
Research Site
Vancouver
British Columbia
Recruiting
Research Site
Toronto
Ontario
Recruiting
Research Site
Beijing
China
Withdrawn
Research Site
Beijing
China
Not Yet Recruiting
Research Site
Beijing
China
Recruiting
Research Site
Changchun
China
Recruiting
Research Site
Changsha
China
Recruiting
+ 63 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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