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

NCT05835310 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: D3461C000302022-502289-25-00
• Study Type: interventional
• Target: 100
• Locations: 17 countries
• Sites: 98

Brief 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)

Conditions

Systemic Lupus Erythematosus

Keywords

Systemic Lupus Erythematosus; SLE; Monoclonal Antibody; Anifrolumab; Parallel-group treatment; Pediatric participants; Standard of care therapy; Intravenous

Outcome Measures

Primary Outcomes (5)

• 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.

  Pre-dose Day 29

• 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.

  Up to Day 29

• 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.

  Up to Day 29

• 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.

  Up to Day 29

• 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 ≥ 1 new BILAG-2004 A or ≥ 2 new BILAG- 2004 B. * No worsening from baseline in 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 PGA 3-point visual analogue scale (VAS).

  At Week 52

Secondary Outcomes (11)

• Part B - Number of participants who are Systemic Lupus Erythematosus Responder Index of ≥ 4 SRI(4) responders (yes/no)

  At Week 52

• Part B - Time to first flare

  Through Week 52

• Part B - Anifrolumab serum concentration

  Pre-dose Week 12, Pre-dose Week 24, Pre-dose Week 52

• Part - B Change from baseline through Week 52 in antidrug antibody (ADA)

  Up to Week 52

• Part - B Change from baseline in anti-double stranded deoxyribonucleic acid antibodies

  At Week 12 and Week 52

Other Outcomes (2)

• All parts - Number of participants reporting suicidal ideation and/or suicidal behavior as per Columbia Suicide Severity Rating Scale (C-SSRS)

  From Week 0 until the follow-up visit (12 weeks post-last dose)

• All parts - Number of participants with adverse events

  From Week 0 until the follow-up visit (12 weeks post-last dose)

Study Arms (2)

Anifrolumab

EXPERIMENTAL

Randomized participants will receive anifrolumab via intravenous (IV) infusion every 4 weeks

Biological: Anifrolumab

Placebo

PLACEBO COMPARATOR

Randomized participants will receive matching placebo via IV infusion

Drug: Placebo

Interventions

Anifrolumab BIOLOGICAL

Participants will receive anifrolumab via IV infusion.

Also known as: (MEDI-546)

Anifrolumab

Placebo DRUG

Participants will receive matching placebo via IV infusion

Placebo

Eligibility Criteria

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

You may qualify if:

• 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.

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.
• 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.

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (98)

Research Site

Phoenix, Arizona, 85016, United States

RECRUITING

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Los Angeles, California, 90027, United States

RECRUITING

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Washington D.C., District of Columbia, 20010, United States

RECRUITING

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Chicago, Illinois, 60611, United States

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Chicago, Illinois, 60637, United States

RECRUITING

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New Orleans, Louisiana, 70118, United States

NOT YET RECRUITING

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Bethesda, Maryland, 20889, United States

WITHDRAWN

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Saint Paul, Minnesota, 55125, United States

RECRUITING

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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

WITHDRAWN

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El Paso, Texas, 79902, United States

RECRUITING

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Houston, Texas, 77030, United States

RECRUITING

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Salt Lake City, Utah, 84108, United States

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Buenos Aires, C1270, Argentina

WITHDRAWN

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Córdoba, 5000, Argentina

RECRUITING

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Rosario, S2000PBJ, Argentina

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San Miguel de Tucumán, T4000AXL, Argentina

NOT YET RECRUITING

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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

NOT YET RECRUITING

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Vancouver, British Columbia, V6H 3N1, Canada

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Toronto, Ontario, M5G 1X8, Canada

RECRUITING

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Beijing, 100020, China

WITHDRAWN

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Beijing, 100032, China

NOT YET RECRUITING

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Beijing, 100730, China

RECRUITING

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Changchun, 130021, China

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Changsha, 410007, China

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Nanjing, 210008, China

NOT YET RECRUITING

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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

WITHDRAWN

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Bordeaux, 33076, France

RECRUITING

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Bron, 69677, France

RECRUITING

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Le Kremlin-Bicêtre, 94275, France

RECRUITING

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Lille, 59037, France

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Toulouse, 31300, France

RECRUITING

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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

NOT YET RECRUITING

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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

RECRUITING

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Yokohama, 232 8555, Japan

RECRUITING

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Yokohama, 236-0004, Japan

RECRUITING

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Atizapán de Zaragoza, 52937, Mexico

NOT YET RECRUITING

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Guadalajara, 44620, Mexico

RECRUITING

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Mérida, 97070, Mexico

RECRUITING

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México, 06720, Mexico

RECRUITING

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Monterrey, 64460, Mexico

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Lodź, 91-738, Poland

RECRUITING

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Warsaw, 02-637, Poland

RECRUITING

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Wroclaw, 52-114, Poland

RECRUITING

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Lisbon, 1169-045, Portugal

WITHDRAWN

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Lisbon, 1649-035, Portugal

WITHDRAWN

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Porto, 4200-319, Portugal

WITHDRAWN

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Cape Town, 7700, South Africa

WITHDRAWN

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Esplugues de Llobregat, 8950, Spain

RECRUITING

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Madrid, 28009, Spain

RECRUITING

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Madrid, 28034, Spain

RECRUITING

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Madrid, 28046, Spain

NOT YET RECRUITING

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Málaga, 29011, Spain

RECRUITING

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Santiago de Compostela, 15706, Spain

NOT YET RECRUITING

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Valencia, 46026, Spain

RECRUITING

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Ankara, 06230, Turkey (Türkiye)

RECRUITING

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Istanbul, 34098, Turkey (Türkiye)

RECRUITING

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Kayseri, 38039, Turkey (Türkiye)

RECRUITING

Research Site

Umraniye, 34760, Turkey (Türkiye)

RECRUITING

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Birmingham, B4 6NH, United Kingdom

RECRUITING

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Bristol, BS2 8BJ, United Kingdom

RECRUITING

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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

RECRUITING

Research Site

Southampton, SO16 6YD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Interventions

anifrolumab

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 18, 2023
• First Posted: April 28, 2023
• Study Start: March 14, 2024
• Primary Completion (Estimated): October 5, 2028
• Study Completion (Estimated): January 9, 2030
• Last Updated: May 6, 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 Pharma Data Sharing Principles. For details of our timelines, please refer 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 de-identified individual patient-level data in an approved sponsored tool. Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at: https://astrazenecagrouptrials.pharmacm.com /ST/Submission/Disclosure.

Locations

AR (4); DE (3); FR (5); PT (3); GB (7); TU (4); ES (7); IT (5); PL (3); US (22); JP (10); CA (3); CO (2); ME (5); ZA (1); CN (10); BR (4)