Systemic Lupus ErythematosusPhase 3BLOSSOMNCT05835310
An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric Participants
A Phase 3 study testing Anifrolumab in people with Systemic Lupus Erythematosus, run by AstraZeneca at 103 sites in 17 countries.
Trial 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) Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Systemic Lupus Erythematosus
- What is being tested?
- Anifrolumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 103 sitesin 17 countries
- Study dates
- 14 Mar 2024to 9 Jan 2030
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
- 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:
- 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.
Who cannot join
- 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.
Show all 11Show fewer
- 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.
Trial locations and contacts
103 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
103 sites in 17 countries
United States22 sites, 20 recruiting
- Withdrawn
Research Site
Bethesda, Maryland, 20889
- Recruiting
Research Site
Chicago, Illinois, 60611
- Recruiting
Research Site
Chicago, Illinois, 60637
- Recruiting
Research Site
Cincinnati, Ohio, 45229
- Recruiting
Research Site
Cleveland, Ohio, 44109
- Recruiting
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Columbus, Ohio, 43203
- Recruiting
Research Site
Durham, North Carolina, 27710
- Recruiting
Research Site
El Paso, Texas, 79902
- Withdrawn
Research Site
Greenville, South Carolina, 29605
- Recruiting
Research Site
Houston, Texas, 77030
- Recruiting
Research Site
Los Angeles, California, 90027
- Recruiting
Research Site
New Hyde Park, New York, 11042
- Recruiting
Research Site
New Orleans, Louisiana, 70118
- Recruiting
Research Site
New York, New York, 10032
- Recruiting
Research Site
Philadelphia, Pennsylvania, 19104
- Recruiting
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Phoenix, Arizona, 85016
- Recruiting
Research Site
Portland, Oregon, 97227
- Recruiting
Research Site
Saint Paul, Minnesota, 55125
- Recruiting
Research Site
Salt Lake City, Utah, 84108
- Recruiting
Research Site
The Bronx, New York, 10467
- Recruiting
Research Site
Valhalla, New York, 10595
- Recruiting
Research Site
Washington D.C., District of Columbia, 20010
China11 sites, 10 recruiting
- Withdrawn
Research Site
Beijing, 100020
- Not yet recruiting
Research Site
Beijing, 100032
- Recruiting
Research Site
Beijing, 100730
- Recruiting
Research Site
Changchun, 130021
- Recruiting
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Changsha, 410007
- Not yet recruiting
Research Site
Dongguan, 523059
- Recruiting
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Nanjing, 210008
- Recruiting
Research Site
Shanghai, 201102
- Recruiting
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Suzhou, 215002
- Recruiting
Research Site
Wenzhou, 325027
- Recruiting
Research Site
Zhengzhou, 450018
Japan11 sites, 11 recruiting
- Recruiting
Research Site
Bunkyō City, 113-8519
- Recruiting
Research Site
Bunkyō City, 113-8603
- Recruiting
Research Site
Chiba, 266-0007
- Recruiting
Research Site
Fuchu-shi, 183-8561
- Recruiting
Research Site
Kawasaki-shi, 216-8511
- Recruiting
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Kobe, 650-0047
- Not yet recruiting
Research Site
Nagasaki, 852-8501
- Recruiting
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Obu-shi, 474-8710
- Recruiting
Research Site
Shinjuku-ku, 162-8666
- Recruiting
Research Site
Yokohama, 232 8555
- Recruiting
Research Site
Yokohama, 236-0004
Spain7 sites, 7 recruiting
- Recruiting
Research Site
Esplugues de Llobregat, 8950
- Recruiting
Research Site
Madrid, 28009
- Recruiting
Research Site
Madrid, 28034
- Recruiting
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Madrid, 28046
- Recruiting
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Málaga, 29011
- Not yet recruiting
Research Site
Santiago de Compostela, 15706
- Recruiting
Research Site
Valencia, 46026
United Kingdom7 sites, 7 recruiting
- Recruiting
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Birmingham, B4 6NH
- Recruiting
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Bristol, BS2 8BJ
- Recruiting
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Liverpool, L12 2AP
- Recruiting
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London, NW1 2PG
- Recruiting
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London, WC1N 3JH
- Recruiting
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Manchester, M13 9WL
- Recruiting
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Southampton, SO16 6YD
Brazil6 sites, 6 recruiting
- Not yet recruiting
Research Site
Belo Horizonte, 30130-100
- Not yet recruiting
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Belo Horizonte, 30150-274
- Recruiting
Research Site
Porto Alegre, 90035-903
- Recruiting
Research Site
Ribeirão Preto, 14048-900
- Recruiting
Research Site
São Paulo, 04024-002
- Recruiting
Research Site
São Paulo, 05403 000
Italy6 sites, 6 recruiting
- Not yet recruiting
Research Site
Florence, 50139
- Recruiting
Research Site
Genova, 16148
- Recruiting
Research Site
Milan, 20122
- Not yet recruiting
Research Site
Milan, 20122
- Recruiting
Research Site
Padova, 35128
- Recruiting
Research Site
Roma, 00165
France5 sites, 5 recruiting
- Recruiting
Research Site
Bordeaux, 33076
- Recruiting
Research Site
Bron, 69677
- Recruiting
Research Site
Le Kremlin-Bicêtre, 94275
- Recruiting
Research Site
Lille, 59037
- Recruiting
Research Site
Toulouse, 31300
Mexico5 sites, 5 recruiting
- Not yet recruiting
Research Site
Atizapán de Zaragoza, 52937
- Recruiting
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Guadalajara, 44620
- Recruiting
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Mérida, 97070
- Recruiting
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México, 06720
- Recruiting
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Monterrey, 64460
Argentina4 sites, 3 recruiting
- Withdrawn
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Buenos Aires, C1270
- Recruiting
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Córdoba, 5000
- Recruiting
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Rosario, S2000PBJ
- Recruiting
Research Site
San Miguel de Tucumán, T4000AXL
Turkey (Türkiye)4 sites, 4 recruiting
- Recruiting
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Ankara, 06230
- Recruiting
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Istanbul, 34098
- Recruiting
Research Site
Kayseri, 38039
- Recruiting
Research Site
Ümraniye, 34760
Canada3 sites, 3 recruiting
- Not yet recruiting
Research Site
Calgary, Alberta, T2N 4N1
- Recruiting
Research Site
Toronto, Ontario, M5G 1X8
- Recruiting
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Vancouver, British Columbia, V6H 3N1
Germany3 sites, 3 recruiting
- Recruiting
Research Site
Berlin, D-13353
- Recruiting
Research Site
Freiburg im Breisgau, 79106
- Recruiting
Research Site
Sankt Augustin, 53757
Poland3 sites, 3 recruiting
- Recruiting
Research Site
Lodź, 91-738
- Recruiting
Research Site
Warsaw, 02-637
- Recruiting
Research Site
Wroclaw, 52-114
Portugal3 sites
- Withdrawn
Research Site
Lisbon, 1169-045
- Withdrawn
Research Site
Lisbon, 1649-035
- Withdrawn
Research Site
Porto, 4200-319
Colombia2 sites, 1 recruiting
- Recruiting
Research Site
Barranquilla, 01800
- Withdrawn
Research Site
Medellín, 050034
About this study
Official title: A 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
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)
Read the 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.
- Conditions
- Systemic Lupus Erythematosus
- Being tested
- Anifrolumab, Placebo
- Keywords
- Systemic Lupus Erythematosus, SLE, Monoclonal Antibody, Anifrolumab, Parallel-group treatment, Pediatric participants, Standard of care therapy, Intravenous
- Medical subject headings
- Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AnifrolumabBiological
Also called (MEDI-546)
Participants will receive anifrolumab via IV infusion.
PlaceboDrug
Participants will receive matching placebo via IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Anifrolumab | Experimental | Randomized participants will receive anifrolumab via intravenous (IV) infusion every 4 weeks Receives: Biological: Anifrolumab |
| Placebo | Placebo comparator | Randomized participants will receive matching placebo via IV infusion Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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 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).
Measured: 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)
SRI-4 response is defined as:
- ≥ 4-point reduction from baseline in SLEDAI-2K score.
- No new organ systems affected as defined by ≥ 1 new BILAG-2004 A or ≥ 2 new BILAG-2004 B items compared to baseline.
- No worsening from baseline in participant's lupus disease activity, defined by an increase ≥ 0.30 points on a PGA 3-point VAS.
Measured: At Week 52
Part B - Time to first flare
Time to first flare, where flare is defined as either ≥ 1 new BILAG-2004 A, or ≥ 2 new BILAG-2004 B items compared with the previous visit.
Measured: Through Week 52
Part B - Anifrolumab serum concentration
The PK of anifrolumab in pediatric participants with moderate to severe active SLE will be characterized.
Measured: Pre-dose Week 12, Pre-dose Week 24, Pre-dose Week 52
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.
Measured: Up to Week 52
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.
Measured: At Week 12 and Week 52
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.
Measured: At Week 12 and Week 52
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.
Measured: At Week 12 and Week 52
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.
Measured: At Week 12 and Week 52
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
Measured: At Week 52
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.
Measured: At Week 52
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.
Measured: At Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2023
- Study start14 Mar 2024
- Primary completion5 Oct 2028 (estimated)
- Study completion9 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus ErythematosusStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05835310
- Sponsor's protocol ID
- D3461C00030
- Other IDs
- 2022-502289-25-00
- Acronym
- BLOSSOM
- Status last verified
- Sep 2026
- First submitted
- 18 Apr 2023
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com /ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.