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Anifrolumab

Phase 3

Cutaneous Lupus Erythematosus | Monoclonal antibody | Immunology |AstraZeneca PLC|Last Updated: Jul 17, 2026

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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment306
FDA Designations
No designations recorded
Clinical trial landscape

Anifrolumab · 21 trials · 17 indications

Phase 3 11Phase 2 6Phase 1 4
NCT07691970Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued TreatmentSystemic Sclerosis Cutaneous Lupus E Myopathies Systemic Lupus Erythematosus
NOT YET_RECRUITING1,072 Analytics
NCT07430306A Study to Evaluate the Treatment Outcomes of Subcutaneous Anifrolumab in Immunosuppressant-naïve and Biologic-naïve Systemic Lupus ErythematosusSystemic Lupus Erythematosus
RECRUITING245 Analytics
NCT06015737A Phase III Study to Investigate the Efficacy and Safety of Anifrolumab in Adults With Chronic and/or Subacute Cutaneous Lupus ErythematosusCutaneous Lupus Erythematosus
ACTIVE NOT_RECRUITING306 Analytics
NCT06455449A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)Polymyositis, Dermatomyositis
RECRUITING240 Analytics
NCT05835310An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric ParticipantsSystemic Lupus Erythematosus
RECRUITING100 Analytics
NCT05925803Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)Systemic Sclerosis
ACTIVE NOT_RECRUITING314 Analytics
NCT05138133Phase 3 Study of Anifrolumab in Adult Patients With Active Proliferative Lupus NephritisLupus Nephritis
ACTIVE NOT_RECRUITING359 Analytics
NCT04931563Anifrolumab Asian PhIII Efficacy Study for Systemic Lupus Erythematosus (SLE)Active Systemic Lupus Erythematosus
COMPLETED277 Analytics
NCT02794285Long Term Safety of Anifrolumab in Adult Subjects With Active Systemic Lupus ErythematosusActive Systemic Lupus Erythematosus
COMPLETED559 Analytics
NCT02446899Efficacy and Safety of Anifrolumab Compared to Placebo in Adult Subjects With Active Systemic Lupus ErythematosusActive Systemic Lupus Erythematosus
COMPLETED373 Analytics
PHASE3NOT YET_RECRUITING
Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued Treatment
Systemic Sclerosis Cutaneous Lupus E Myopathies Systemic Lupus ErythematosusUnlock trial analytics
PHASE3RECRUITING
A Study to Evaluate the Treatment Outcomes of Subcutaneous Anifrolumab in Immunosuppressant-naïve and Biologic-naïve Systemic Lupus Erythematosus
Systemic Lupus ErythematosusUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase III Study to Investigate the Efficacy and Safety of Anifrolumab in Adults With Chronic and/or Subacute Cutaneous Lupus Erythematosus
Cutaneous Lupus ErythematosusUnlock trial analytics
PHASE3RECRUITING
A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)
Polymyositis, DermatomyositisUnlock trial analytics
PHASE3RECRUITING
An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric Participants
Systemic Lupus ErythematosusUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)
Systemic SclerosisUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Phase 3 Study of Anifrolumab in Adult Patients With Active Proliferative Lupus Nephritis
Lupus NephritisUnlock trial analytics
PHASE3COMPLETED
Anifrolumab Asian PhIII Efficacy Study for Systemic Lupus Erythematosus (SLE)
Active Systemic Lupus ErythematosusUnlock trial analytics
PHASE3COMPLETED
Long Term Safety of Anifrolumab in Adult Subjects With Active Systemic Lupus Erythematosus
Active Systemic Lupus ErythematosusUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Anifrolumab Compared to Placebo in Adult Subjects With Active Systemic Lupus Erythematosus
Active Systemic Lupus ErythematosusUnlock trial analytics
Study Endpoints
Primary Endpoints
SAEs and Non-Serious AEs
From first dose administered and until 12 weeks after the last dose of Anifrolumab

Safety will be evaluated by monitoring and assessing SAEs and Non-Serious AEs as reported throughout the study and until 12 weeks after the last dose of anifrolumab

Attainment of DORIS remission
At Week 52

Proportion of participants who are in DORIS remission at Week 52 will be assessed. DORIS remission is defined as Clinical SLEDAI-2K (sum of all SLEDAI-2K items except for increased deoxyribonucleic acid \[DNA\] binding and low complement) = 0; PGA \[0-3\] \< 0.5, prednisone 5 mg/day or less, and stable antimalarials, ISs, and biologics.

Number of participants with a 70% reduction relative to baseline in the Cutaneous Lupus Erythematosus Disease Area and Severity Index-Activity (CLASI-A) score
At Week 24

The CLASI is a validated index used for assessing the cutaneous lesions of SLE. It consists of 2 separate scores: i) activity of the disease, and ii) measure of damage. The CLASI instrument will be used at each study visit to capture individual skin manifestation scores. CLASI-70 responder (Yes/No) is defined as a participant who achieves a 70% reduction relative to baseline in CLASI-A score. Otherwise, the participant is considered a non-responder.

Total Improvement Score (TIS) ≥ 40 response
52 week

Participants who have at least moderate improvement in disease activity TIS ≥ 40 and has not met "confirmed deterioration" criteria at 2 consecutive visits

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).

Number of participants responding to treatment based on the Revised Composite Response Index in Systemic Sclerosis (CRISS-25)
at Week 52

Number of participants meeting all the criteria: * Improvement in at least 2 components (≥5% increase for percent predicted Forced Vital Capacity (FVC) and/or≥25% decrease for Modified Rodnan Skin Score (mRSS), Health Assessment Questionnaire Disability Index (HAQ-DI), Patient Global Assessment (PtGA), Clinician Global Assessment (CGA) * Worsening in no more than one component (≥5% decrease percent predicted FVC and/or≥25% increase for mRSS, HAQ-DI, PtGA, CGA) * No significant SSc-related event as defined by: New scleroderma renal crisis New decline in percent predicted FVC≥15% in established interstitial lung disease or new percent predicted FVC below 80% predicted New onset of left ventricular failure requiring treatment New onset of pulmonary arterial hypertension requiring treatment Gastrointestinal dysmotility requiring enteral or parenteral nutrition Digital ischemia with gangrene, amputation, or hospitalization requiring treatment -Otherwise, a participant is a non-responder

Difference in proportion of participants with CRR (Complete Renal Response) in anifrolumab group compared with placebo group
Week 52

CRR is defined as: * UPCR ≤ 0.5 mg/mg * eGFR ≥ 60 mL/min/1.73 m2 or no decrease from baseline of ≥ 20%

Difference in Proportion of Participants Who Are Responders Between Anifrolumab and Placebo
Week 52

Composite endpoint (BICLA),a composite binary endpoint defined by meeting all of the following criteria: * Reduction of all baseline BILAG-2004 A to B/C/D and baseline BILAG-2004 B to C/D, and no BILAG-2004 worsening in other organ systems, where worsening is defined as ≥1 new BILAG-2004 A or ≥2 new BILAG-2004 B * No worsening from baseline in SLEDAI-2K, where worsening is defined as an increase from baseline of \>0 points in SLEDAI-2K * No worsening from baseline in participants' lupus disease activity, where worsening is defined as an increase of ≥0.30 points on a 3-point PGA visual analogue scale (VAS)

Exposure-adjusted Incidence Rates (EAIRs) of Adverse Events of Special Interest (AESIs)
Up to a maximum of 1114 days

The event rate per 100 participant years was defined as the number of participants with an event divided by the sum of exposure time during the LTE study (including follow-up) in days for all participants in the analysis set multiplied by 365.25 days/year multiplied by 100. The exposure in a time period for each participant was calculated as end of period - start of period + 1. EAIRs of AESIs are presented as event rate per 100 participant years. The following AESIs were pre-defined: * Non-opportunistic serious infections * Opportunistic infections * Anaphylaxis * Malignancy * Herpes zoster * Tuberculosis (TB) (including latent TB) * Influenza * Vasculitis (non-systemic lupus erythematosus \[SLE\]) * Major cardiovascular events as according to the Cardiovascular Event Adjudication Committee.

EAIRs of Serious Adverse Events (SAEs)
Up to a maximum of 1114 days

EAIRs of SAEs are presented as event rate per 100 participant years. An SAE was an AE occurring during any study phase that fulfils 1 or more of the following criteria: * Results in death * Is immediately life-threatening * Requires in-patient hospitalisation or prolongation of existing hospitalisation * Results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions * Is a congenital abnormality or birth defect * Is an important medical event that may jeopardise the participant or may require medical intervention to prevent one of the outcomes listed above.

Number of Participants Who Achieved the British Isles Lupus Assessment Group Based Composite Lupus Assessment (BICLA) Response at Week 52
Baseline; Week 52

Composite endpoint BICLA was defined by meeting all of the following criteria: * Reduction of all baseline British Isles Lupus Assessment Group (BILAG)-2004 A to B/C/D and baseline BILAG-2004 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), where worsening is defined as an increase from baseline of \>0 points in SLEDAI-2K * No worsening from baseline in participants' lupus disease activity, where worsening is defined by an increase ≥0.30 points on a 3-point Physician's Global Assessment (PGA) visual analogue scale (VAS) * No discontinuation of investigational product * No use of restricted medications beyond the protocol allowed threshold before assessment

Number of Participants Who Achieved a Systemic Lupus Erythematosus (SLE) Responder Index ≥4 (SRI[4]) at Week 52 (Original Analysis With Restricted Medication Rules)
Week 52

SRI(4) was defined as meeting all of the following criteria: Reduction from baseline of ≥4 points in the Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) No new organ systems affected, defined by 1 or more British Isles Lupus Assessment Group (BILAG-2004) A or 2 or more BILAG-2004 B items No worsening from baseline in participants lupus disease activity. Worsening was defined as an increase of ≥0.30 points on a 3-point Physician's Global Assessment (PGA) visual analogue scale (VAS) No discontinuation of investigational product and no use of restricted medications beyond the pre-specified analysis threshold.

Percentage of participants achieving at least a Hidradenitis Suppurativa Clinical Response score of 50 (HiSCR 50) at 12 weeks
12 weeks

The HiSCR is a validated assessment tool developed for patients with hidradenitis suppurativa. It is a dichotomized clinical response assessment that measures the status of three types of lesions: abscesses, inflammatory nodules, and draining fistulas. HiSCR 50 is defined as participants who have at least a 50% reduction in abscess and nodule count compared to baseline and no increase in the number of abscesses or draining tunnels.

Maximum observed serum (peak) concentration (Cmax)
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

To characterize PK (Cmax) of anifrolumab in pediatric participants with SLE following SC administration.

Area under the serum concentration-time curve (AUC)
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

To characterize PK (AUC) of anifrolumab in pediatric participants with SLE following SC administration.

Time to maximum plasma concentration (tmax)
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

To characterize PK (tmax) of anifrolumab in pediatric participants with SLE following SC administration.

Apparent total body clearance of drug from plasma (CL/F)
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11

To characterize PK (CL/F) of anifrolumab in pediatric participants with SLE following SC administration.

Trough drug concentration at steady state (Ctrough,ss)
At Week 12

To characterize PK (Ctrough,ss) of anifrolumab in pediatric participants with SLE following SC administration.

Maximum Concentration of Anifrolumab in Serum After First Dose
Week 0

Maximum concentration (Cmax) of anifrolumab is based on sample collected 5 to 8 days after the first dose of strudy treatment.

Steady-state Serum Trough (Predose) Concentration (Ctrough) of Anifrolumab
Week 12

Steady-state serum through concentration (Ctrough) is based on sample collected at Week 12 prior to dosing of study treatment (predose).

21-gene Type 1 IFN Signature Score (Fold-change)
Week 12

21-gene type I IFN signature score (fold change) is based on samples collected both at baseline and Week 12 prior to dosing of study treatment. Levels of 21-gene type I IFN pharmacodynamics signature is derived as relative to a pooled normal control.

21-gene Type 1 IFN Neutralization Ratio (Percent Suppression of Fold Change)
Week 12

21-gene type I IFN signature score (fold change) is based on samples collected both at baseline and Week 12 prior to dosing of study treatment. For each individual participant and assessment, the level of 21-gene type I IFN pharmacodynamics signature is derived as relative to a pooled normal control, as the median of 100-(((baseline-Week 12)/baseline)\*100) for the 21 genes. At a population level, the results are presented as mean the above.

Change From Baseline in 24-hour Urine Protein to Creatinine Ratio (UPCR)
From Week 1 (Baseline) up to Week 52

To evaluate the efficacy of anifrolumab plus SOC (combination of mycophenolate mofetil and corticosteroids) compared with placebo plus SOC in subjects with active proliferative lupus nephritis (LN). Geometric mean ratio of 24-hour UPCR at week 52 over baseline. Values \<1 indicate improvement from baseline.

Number of Particpants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
From first dose of study drug (Day 1) through 168 weeks

An adverse event (AE) is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug.

Number of Participants With Adverse Events Resulting in Discontinuation (DAEs) of Anifrolumab
From first dose of study drug (Day 1) through 168 weeks

Number of participants with DAEs are reported.

Number of Participants With Adverse Events of Special Interest (AESIs)
From first dose of study drug (Day 1) through 168 weeks

An AESI is scientific and medical concern specific to understanding of the study drug. An AESI may be serious or non-serious. Number of participants with AESIs are reported.

Percentage of Participants Achieving an Systemic Lupus Erythematosus (SLE) Responder Index [SRI (4)] Response With Oral Corticosteroids (OCS) Tapering at Day 169
Day 169

An SRI (4) responder defined as a participant who had 1) a reduction in baseline Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score of greater than or equal to (\>=) 4 points; 2) no worsening of disease from baseline as measured by the Physician Global Assessment (MDGA) (worsening was defined as an increase of \>= 0.3 from baseline on a 0 to 3.0 visual analog scale); and 3) no new British Isles Lupus Assessment Group 2004 (BILAG-2004) Index 'A' organ system score and no more than one new or worsening BILAG-2004 Index 'B' organ system score. OCS tapering requires a sustained reduction of OCS from Day 85 through Day 169 \[less than 10 milligram per day (mg/day) and less or equal to the dose received on Day 1\]. SRI was analyzed by a logistic regression model.

Percentage of Type I Interferon (IFN) Test High Participants Achieving an Systemic Lupus Erythematosus Responder Index (SRI) (4) Response With Oral Corticosteroids (OCS) Tapering at Day 169
Day 169

Type I IFN signature in whole blood assessed by using a 4-gene diagnostic test. The blood samples collected were to be used to prospectively identify participants as IFN test-high or test-low. The results of this test were used to stratify participants. An SRI (4) Responder was defined as a participant who had 1) a reduction in baseline SLEDAI-2K disease activity score of \>= 4 points; 2) no worsening of disease from baseline as measured by the Physician Global Assessment (MDGA) (worsening was defined as an increase of \>= 0.3 from baseline on a 0 to 3.0 visual analog scale); and 3) no new British Isles Lupus Assessment Group 2004 (BILAG-2004) Index A organ system score and no more than one new or worsening BILAG-2004 Index B organ system score. OCS tapering requires a sustained reduction of OCS from Day 85 through Day 169 \[less than 10 mg/day and less or equal to the dose received on Day 1\]. SRI was analyzed by a logistic regression model.

SC and IV Arm: Area under the serum concentration-time curve from the pre-dose concentration extrapolated to infinity (AUCinf)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (AUCinf will be derived).

SC and IV Arm: Maximum observed serum (peak) concentration (Cmax)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (Cmax will be derived).

SC and IV Arm: Time to reach peak or maximum observed concentration (tmax)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (tmax will be derived).

SC and IV Arm: Area under the serum concentration-time curve from pre-dose concentration to time of last quantifiable concentration (AUClast)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (AUClast will be derived).

SC and IV Arm: half-life associated with the terminal slope of a semi-logarithmic concentration-time curve (t½λz)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (t½λz will be derived).

Bioavailability (F)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (F will be derived).

SC Arm: Apparent total body clearance of drug after extravascular administration (CL/F)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (CL/F will be derived).

SC Arm:Volume of distribution during the terminal phase after extravascular administration (Vz/F)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (Vz/F will be derived).

IV Arm: volume of distribution during the terminal phase after intravenous administration (Vz)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (Vz will be derived).

IV Arm: Apparent total body clearance of drug after intravenous administration (CL)
At predefined intervals throughout the study period (From Day 1 to Day 57)

The concentration of anifrolumab in serum will be determined (CL will be derived).

Area under serum concentration-time curve from time zero extrapolated to infinity (AUCinf)
Up to Day 57

Evaluation of AUCinf following single SC administration of anifrolumab by AI is comparable to the AUCinf following single SC administration of anifrolumab using APFS will be done.

Area under serum concentration-time curve from time zero to last quantifiable concentration (AUClast)
Up to Day 57

Evaluation of AUClast following single SC administration of anifrolumab by AI is comparable to the AUClast following single SC administration of anifrolumab using APFS will be done.

Maximum observed serum (peak) drug concentration (Cmax)
Up to Day 57

Evaluation of Cmax following single SC administration of anifrolumab by AI is comparable to the Cmax following single SC administration of anifrolumab using APFS will be done.

Time to maximum observed plasma concentration (Tmax) of anifrolumab.
Day 1 to Day 141

To characterise the pharmacokinetic (PK) profile of anifrolumab via intravenous (IV) infusion.

Maximum observed plasma concentration (Cmax) of anifrolumab.
Day 1 to Day 141

To characterise the pharmacokinetic (PK) profile of anifrolumab via intravenous (IV) infusion.

Area under plasma concentration-time curve over dosing interval (AUC[tau]) of anifrolumab.
Day 1 to Day 141

To characterise the pharmacokinetic (PK) profile of anifrolumab via intravenous (IV) infusion.

Pre-dose trough concentration (Ctrough) of anifrolumab.
Day 1 to Day 141

To characterise the pharmacokinetic (PK) profile of anifrolumab via intravenous (IV) infusion.

The volume of plasma cleared of drug per unit time (CL) of anifrolumab.
Day 1 to Day 141

To characterise the pharmacokinetic (PK) profile of anifrolumab via intravenous (IV) infusion.

Pharmacokinetics: Observed Maximum Serum Concentration (Cmax) Following Single Dose of Anifrolumab.
On Day 1 pre-dose and at 5 minutes (IV cohort only), 24 and 48 hours post-dose and at each follow-up visit, up to 85 days

To evaluate Cmax of anifrolumab after single administration of two doses subcutaneously and one dose intravenously. Up to 13 blood samples were collected in total.

Pharmacokinetics: Area Under the Serum Concentration-time Curve From Time Zero to Time of Last Quantifiable Concentration (AUC0-t) Following Single Dose of Anifrolumab
On Day 1 pre-dose and at 5 minutes (IV cohort only), 24 and 48 hours post-dose and at each follow-up visit, up to 85 days

To evaluate AUC(0-t) of anifrolumab after single administration of two doses subcutaneously and one dose intravenously Up to 13 blood samples were collected in total.

Pharmacokinetics: Area Under Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC) Following Single Dose of Anifrolumab
On Day 1 pre-dose and at 5 minutes (IV cohort only), 24 and 48 hours post-dose and at each follow-up visit, up to 85 days

To evaluate AUC of anifrolumab after single administration of two doses subcutaneously and one dose intravenously. Up to 13 blood samples were collected in total.

Safety: Number of Participants With Adverse Events (AEs)
From screening to final follow-up visit, up to 16 weeks

To assess the safety and tolerability of single doses of anifrolumab

Safety: Summary of Local Injection Site Pain (SC Cohorts) Assessed in Participants
Immediately after dosing, at 10, 20 minutes and 1 hour after injection

Local injection site pain was assessed using a 100 mm participant rated Visual Analog Scale (VAS 0mm - 100mm ungraduated scale, where 0 = "no pain" to 100 = "worst imaginable pain"). This assessment was taken for only those participants in subcutaneously dosed treatment groups; 600 mg SC and 300 mg SC, anifrolumab and placebo. For the 300 mg SC anifrolumab and 300 mg SC placebo groups which received two simultaneous injections, the average VAS score (0mm-100mm) of the two injection sites were reported.

Safety: Summary of Local Injection Site Pruritus (SC Cohorts) Assessed in Participants
Immediately after dosing, at 10, 20 minutes and 1 hour after injection

Local injection site pruritus was assessed using a 100 mm participant rated Visual Analog Scale (VAS 0mm - 100mm ungraduated scale, where 0 = "no itching" to 100 = "worst imaginable itching"). This assessment was taken for only those participants in subcutaneously dosed treatment groups; 600 mg SC and 300 mg SC, anifrolumab and placebo. For the 300 mg SC anifrolumab and 300 mg SC placebo groups which received two simultaneous injections, the average VAS score (0mm-100mm) of the two injection sites were reported.

Safety: Summary of Erythema Injection Site Reaction (SC Cohorts) Assessed in Participants
Immediately after dosing, at 10, 20 minutes and 1 hour after injection

Erythema was measured as the largest diameter across the needle site on the skin in millimetres (mm). This assessment was taken for only those participants in subcutaneously dosed treatment groups; 600 mg SC and 300 mg SC, anifrolumab and placebo. For the 300 mg SC anifrolumab and 300 mg SC placebo groups which received two simultaneous injections, the average of the two injection site diameters (mm) were reported.

Safety: Summary of the Induration Injection Site Reaction (SC Cohorts) Assessed in Participants
Immediately after dosing, at 10, 20 minutes and 1 hour after injection

Induration was measured as the largest diameter across the needle site on the skin in millimetres (mm). This assessment was taken for only those participants in subcutaneously dosed treatment groups; 600 mg SC and 300 mg SC, anifrolumab and placebo. For the 300 mg SC anifrolumab and 300 mg SC placebo groups which received two simultaneous injections, the average of the two injection site diameters (mm) were reported.

Secondary Endpoints
To assess the attainment of low level disease activity
At Week 28 and 52
Time spent in DORIS remission, LLDAS or LLDAS-5
At Week 52
Sustaining DORIS remission, LLDAS or LLDAS-5
All subsequent visits including 52 Week
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AnifrolumabEXPERIMENTALThis is a single arm open-label study with the primary objective of providing continued access to anifrolumab to eligible participants who are continuing to derive clinical benefit from treatment and assessing long-term safety and tolerability of anifrolumab until participant meets one of the study discontinuation criteria.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo as a SC injection from Week 0/Day 1 up to Week 23. From Week 24 the participants will receive anifrolumab up to and including Week 51.
Anifrolumab (subcutaneous weekly injection)EXPERIMENTALAnifrolumab subcutaneous injection once weekly
Placebo (subcutaneous weekly injection)PLACEBO_COMPARATORMatched placebo control subcutaneous injection once weekly
matched placebo control (subcutaneous weekly injection)PLACEBO_COMPARATORmatched placebo control subcutaneous injection once weekly
Anifrolumab - higher doseEXPERIMENTALAnifrolumab
Anifrolumab - lower doseEXPERIMENTALAnifrolumab
Cohort 1 (body weight > 40 kg)EXPERIMENTALParticipants with body weight \> 40 kg will receive anifrolumab as an injection in APFS.
Cohort 2 (body weight ≥ 15 to ≤ 40 kg)EXPERIMENTALParticipants with body weight ≥ 15 to ≤ 40 kg will receive anifrolumab as an injection in APFS.
Placebo matching for lower dose of AnifrolumabPLACEBO_COMPARATOR1ml, once every second week, one subcutaneous injection added to stand of care, from week 0 to week 50
Placebo matching for higher dose of AnifrolumabPLACEBO_COMPARATOR2×1ml , once every second week, two subcutaneous injections as added to stand of care, from week 0 to week 50
Anifrolumab (MEDI-546) 300 mgEXPERIMENTALParticipants will receive 300 milligram (mg) anifrolumab as an intravenous infusion every 4 weeks for 48 weeks.
Anifrolumab (MEDI-546) 1000 mgEXPERIMENTALParticipants will receive 1000 mg anifrolumab as an intravenous infusion every 4 weeks for 48 weeks.
Matching PlaceboPLACEBO_COMPARATORParticipants will receive placebo matched to anifrolumab intravenous (IV) infusion every 4 weeks for 48 weeks.
SubcutaneousEXPERIMENTAL -
IntravenousEXPERIMENTAL -
Anifrolumab administered using AIEXPERIMENTALRandomized participants will receive a single SC dose of anifrolumab via AI.
Anifrolumab administered using APFSACTIVE_COMPARATORRandomized participants will receive a single SC dose of anifrolumab via APFS.
Anifrolumab 300 mg SC injectionsEXPERIMENTAL300 mg single dose anifrolumab delivered as 2 separate 1 mL SC injections administered serially
Anifrolumab 300 mg IV infusionEXPERIMENTAL300 mg single dose anifrolumab delivered as an IV infusion over 30 minutes
Anifrolumab 600 mg SC infusionEXPERIMENTAL600 mg single dose anifrolumab or placebo delivered as 4 mL SC by infusion pump
Placebo 300 mg SC injectionsPLACEBO_COMPARATOR300 mg single dose placebo delivered as 2 separate 1 mL SC injections administered serially
Placebo 300 mg IV infusionPLACEBO_COMPARATOR300 mg single dose placebo delivered as an IV infusion over 30 minutes
Placebo 600mg SC infusionPLACEBO_COMPARATOR600 mg single dose placebo delivered as 4 mL SC by infusion pump
Interventions
NameTypeDescription
AnifrolumabCOMBINATION_PRODUCTAnifrolumab pre-filled syringe
PlaceboOTHERMatching placebo solution for injection in aPFS.
Anifrolumab (blinded)COMBINATION_PRODUCTAnifrolumab treatment delivered subcutaneously, once weekly for 52 weeks
Anifrolumab (unblinded, open label)COMBINATION_PRODUCTAt Week 52, all study participants on anifrolumab or placebo will receive open label anifrolumab once weekly for an additional 52 weeks
Placebo (blinded)DRUGmatched placebo delivered subcutaneously, once weekly for 52 weeks
Anifrolumab + APFSCOMBINATION_PRODUCTAnifrolumab will be administered as a SC injection using an APFS.
Anifrolumab 300 mgBIOLOGICALParticipants will receive 300 milligram (mg) anifrolumab as an intravenous infusion every 4 weeks for 48 weeks.
Anifrolumab 1000 mgBIOLOGICALParticipants will receive 1000 mg anifrolumab as an intravenous infusion every 4 weeks for 48 weeks.
AutoinjectorDEVICEAutoinjector will be use to administer single SC dose of anifrolumab.
Accessorized Pre-Filled SyringeDEVICEAccessorized Pre-filled syringe will be use to administer single SC dose of anifrolumab.
Anifrolumab SC injection (300mg)DRUG300 mg of anifrolumab delivered as 2 separate 1 mL SC injections administered serially on Day 1
Anifrolumab IV infusion (300mg)DRUG300 mg of anifrolumab delivered as an IV infusion over 30 minutes on Day 1
Anifrolumab SC infusion (600mg)DRUG600 mg of anifrolumab delivered as 4 mL SC by infusion pump on Day 1
Anifrolumab placebo SC injection (300mg)DRUG300mg of placebo delivered as 2 separate 1 mL SC injections administered serially on Day 1
Anifrolumab placebo IV infusion (300mg)DRUG600mg of placebo delivered as an IV infusion over 30 minutes on Day 1
Anifrolumab placebo SC infusion (600mg)DRUG600 mg of placebo delivered as 4 mL SC by infusion pump on Day 1
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Eligibility Criteria
Age Range5 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria: * Participants are eligible to be included in the study only if all of the following criteria apply: 1 Participants must have completed the entirety of the parent study and should have received at least one dose of anifrolumab in the parent study. * In the opinion of the Inve...

Countries:United StatesAustriaBelgiumBrazilBulgariaFranceGreeceIndiaIsraelItalyJapanPhilippinesPolandRomaniaSerbiaSlovakiaSouth KoreaSpainTaiwanThailandTurkey (Türkiye)United KingdomCanadaGermanyMexicoArgentinaAustraliaChileChinaColombiaDenmarkNetherlandsNew ZealandPortugalSouth AfricaCzechiaHungaryPuerto RicoSwedenVietnamMalaysiaPeruRussiaHong KongLithuaniaUkraine
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