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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03656562
Registration number
NCT03656562
Ethics application status
Date submitted
19/07/2018
Date registered
4/09/2018
Date last updated
25/06/2024
Titles & IDs
Public title
Study the Efficacy and Safety of VAY736 and CFZ533 in SLE Patients
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Scientific title
A Placebo-controlled, Patient and Investigator Blinded, Randomized Parallel Cohort Study to Assess Pharmacodynamics, Pharmacokinetics, Safety, Tolerability and Preliminary Clinical Efficacy of VAY736 and CFZ533 in Patients With Systemic Lupus Erythematosus (SLE)
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Secondary ID [1]
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2018-001508-12
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Secondary ID [2]
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CVAY736X2208
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Systemic Lupus Erythematosus (SLE)
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - VAY736
Treatment: Drugs - VAY736 Placebo
Treatment: Drugs - CFZ533
Treatment: Drugs - CFZ533 Placebo
Experimental: Cohort 1 VAY736 - Blinded treatment phase:
VAY736 administered subcutaneously (s.c.) every 4 weeks as multiple doses of VAY736 150 mg (total dose being VAY736 300 mg) until Week 25 + Standard of Care (SoC) for systemic lupus erythematosus (SLE).
Open-label treatment phase:
VAY736 administered subcutaneously (s.c.) every 4 weeks as multiple doses of VAY736 150 mg (total dose being VAY736 300 mg) until Week 49.
Placebo comparator: Cohort 1 VAY736 Placebo - Blinded treatment phase:
VAY736 matching placebo administered subcutaneously (s.c.) every 4 weeks as multiple doses of placebo 0 mg until Week 25 + Standard of Care (SoC) for systemic lupus erythematosus (SLE).
Open-label treatment phase:
VAY736 administered subcutaneously (s.c.) every 4 weeks as multiple doses of VAY736 150 mg (total dose being VAY736 300 mg) until Week 49.
Experimental: Cohort 2 CFZ533 - Blinded treatment phase:
CFZ533 administered intravenously (i.v) every 4 weeks as multiple doses of CFZ533 150 mg, based on body weight (BW) of the patients (10 mg/kg (\>= 50 kg BW) and 13 mg/kg (\< 50 kg BW)) until Week 25 + Standard of Care (SoC) for systemic lupus erythematosus (SLE).
Open-label phase:
CFZ533 administered intravenously (i.v) every 4 weeks as multiple doses of CFZ533 150 mg, based on body weight (BW) of the patients (10 mg/kg (\>= 50 kg BW) and 13 mg/kg (\< 50 kg BW)) until Week 49.
Placebo comparator: Cohort 2 CFZ533 Placebo - Blinded treatment phase:
CFZ533 matching placebo administered intravenously (i.v) every 4 weeks as multiple doses of placebo 0 mg, based on body weight (BW) of the patients (10 mg/kg (\>= 50 kg BW) and 13 mg/kg (\< 50 kg BW)) until Week 25 + Standard of Care (SoC) for systemic lupus erythematosus (SLE).
Open-label phase:
CFZ533 administered intravenously (i.v) every 4 weeks as multiple doses of CFZ533 150 mg, based on body weight (BW) of the patients (10 mg/kg (\>= 50 kg BW) and 13 mg/kg (\< 50 kg BW)) until Week 49.
Treatment: Drugs: VAY736
150 mg powder in vial for solution for injection; after reconstitution to 150 mg/mL per vial, a dose of 300 mg
Treatment: Drugs: VAY736 Placebo
solution for injection; 0 mg/mL administered as 2 mL s.c. injection
Treatment: Drugs: CFZ533
150 mg/mL as concentrate in vial for infusion, administered at a dose of 10 mg/kg as i.v. infusion
Treatment: Drugs: CFZ533 Placebo
Placebo as concentrate in vial for infusion, administered at a dose of 10 mg/kg as i.v. infusion
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Participants With SLE Responder Index (SRI)-4 Response Status at Week 29 With Reduced Steroid Dose Maintained Between Weeks 17 and 29
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Assessment method [1]
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The primary endpoint is a composite of SRI-4 response at Week 29 with sustained reduction in oral corticosteroid from Week 17 through Week 29. Patients taking other rescue medication or prohibited medication or drop out before Week 29 were considered non-responders.
SRI-4 response is defined as below:
* having \>= 4 points reduction from baseline in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score (score is 0 to 105; a higher score indicating more severe disease) AND
* no new British Isles Lupus Activity Group (BILAG)-2004 A organ domain score and no more than one new BILAG-2004 B organ domain scores compared with baseline AND
* \<10 mm point increase from baseline with scale 0 to 100 mm in the physician's global assessment from baseline
Sustained reduction in oral corticosteroid is defined as below:
* =\< 5 mg/day or less than or equal to baseline dose, whichever was lower at Week 17 AND
* no increase of that dose from Week 17 through Week 29
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Timepoint [1]
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Baseline, Week 17 to Week 29
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Secondary outcome [1]
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Changes Between Baseline and Week 29 in the Physicians' Global Assessment (PhGA) Visual Analog Scale (VAS) Assessing Patient's Overall Disease Activity
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Assessment method [1]
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The Physician's global assessment (PhGA-VAS) of disease activity was performed using 100 mm VAS ranging from "no disease activity" (score 0) to "maximal disease activity" (score 100), after the question on how well the patient was doing with the disease considering all aspects affected by the disease. The investigator was then measuring the distance in mm from the left edge of the scale and entering the value.
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Timepoint [1]
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Baseline, Week 5, Week 9, Week 13, Week 17, Week 21, Week 25, Week 29
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Secondary outcome [2]
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Changes Between Baseline and Week 29 in the Patient's Global Assessment (PGA) Visual Analog Scale (VAS) Assessing Patient's Global Disease Activity
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Assessment method [2]
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The patient's global assessment of disease activity was performed using a Visual Analogue Scale (VAS) of 100 mm ranging from "no disease activity" (score 0) to "severe disease activity" (score 100), after the question on how well the patient was doing with the disease considering all aspects affected by the disease. The investigator was then measuring the distance in mm from the left edge of the scale and entering the value.
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Timepoint [2]
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Baseline, Week 5, Week 9, Week 13, Week 17, Week 21, Week 25, Week 29
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Secondary outcome [3]
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Flare Rate and Time to First Flare
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Assessment method [3]
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Flare rate and time to first flare, with flare defined as one new 'A' score or two or more 'B' score using BILAG -2004
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Timepoint [3]
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18 months
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Secondary outcome [4]
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Time to First Flare
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Assessment method [4]
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Time to first flare, with flare defined as one new 'A' score or two or more 'B' score using BILAG -2004
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Timepoint [4]
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18 months
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Secondary outcome [5]
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PK Cohort 1 - Cmax,ss
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Assessment method [5]
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PK Cohort 1 (VAY736): free VAY736 serum concentration (Cmax at steady state)
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Timepoint [5]
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18+ months
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Secondary outcome [6]
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PK Cohort 1 - Ctrough,ss
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Assessment method [6]
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PK Cohort 1 (VAY736): free VAY736 serum concentration (Ctrough at steady state)
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Timepoint [6]
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18+ months
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Secondary outcome [7]
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PK Cohort 2 - Cmax,ss
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Assessment method [7]
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PK Cohort 2 (CFZ533): free CFZ533 concentration in plasma (Cmax at steady state).
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Timepoint [7]
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18 months
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Secondary outcome [8]
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PK Cohort 2 - Ctrough,ss
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Assessment method [8]
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PK Cohort 2 (CFZ533): free CFZ533 concentration in plasma (Ctrough at steady state).
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Timepoint [8]
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18 months
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Secondary outcome [9]
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PD Cohort 2 (CFZ533): Total Soluble CD40
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Assessment method [9]
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PD Cohort 2 (CFZ533): total soluble CD40 in plasma.
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Timepoint [9]
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18 months
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Eligibility
Key inclusion criteria
* Written informed consent must be obtained before any assessment is performed
* Fulfill =4 of the 11 American College of Rheumatology 1997 classification criteria for SLE
* Patient diagnosed with SLE for at least 6 months prior to screening
* Elevated serum titers at screening of ANA (=1:80) of a pattern consistent with an SLE diagnosis, including at a minimum either anti-double stranded DNA (anti-ds DNA) or anti-Ro (SSA) or anti-La (SSB) or anti-nuclear ribonucleoprotein (anti-RNP) or anti-Smith (anti-Sm)
* Currently receiving corticosteroids and/or anti-malarial and/or thalidomide treatment and/or another DMARD on a stable dose according to protocol requirements
* SLEDAI-2K score of =6 at screening
* BILAG 2004 score of one "A" score either in the mucocutaneous or in the musculoskeletal domain or one "B" score in either the mucocutaneous or musculoskeletal domain and at least one "A" or "B" score in a second domain at screening
* Weigh at least 40 kg at screening
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Minimum age
18
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Maximum age
75
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Cohort 2 (CFZ533/Placebo) only:
* Patients who are at significant risk for thromboembolic events based on the following:
* History of either thrombosis or 3 or more spontaneous abortions
* Presence of lupus anticoagulant or significantly prolonged activated partial thromboplastin time (aPTT) consistent with co-existent anti-phospholipid syndrome and without concurrent prophylactic treatment with aspirin or anticoagulants as per local standard of care
All Cohorts:
* History of receiving prior to screening:
* Within 12 weeks: i.v. corticosteroids, calcineurin inhibitors or other oral DMARD
* Within 24 weeks: cyclophosphamide or biologics such as intravenous Ig, plasmapheresis, anti-TNF-a mAb, CTLA4-Fc Ig (abatacept) or BAFF targeting agents (e.g., belimumab)
* Any B-cell depleting therapies (e.g., anti-CD20 mAb, anti-CD22 mAb, anti-CD52 mAb) or TACI-Ig (atacicept) administered within 52 weeks prior to screening, and a B-cell count <50 cells/µ at the time of screening
* Evidence of past exposure to tuberculosis as assessed by Quantiferon testing at screening
* Presence of human immunodeficiency virus (HIV) infection at screening
* Severe organ dysfunction or life threatening disease; ECOG performance status > 1 at screening
* Presence of WHO Class III-IV renal involvement with proliferative disease Presence of severe lupus kidney disease as defined by proteinuria above 6 g/day or equivalent using spot urine protein creatinine ratio, or serum creatinine greater than 2.5 mg/dL (221.05 µmol/L), or requiring immune suppressive induction or maintenance treatment exceeding protocol defined limits
* Active viral, bacterial or other infections at the time of screening or enrollment
* Receipt of live/attenuated vaccine within a 2-month period before first dosing
* Uncontrolled, co-existing serious disease, e.g., uncontrolled hypertension, heart failure, type I diabetes, thyroid disease within 3 months prior to first dosing, or significant, unresolved illness within 2 weeks prior to first dosing
* History of hypersensitivity to drugs of similar chemical class
* Chronic infection with hepatitis B (HBV) or hepatitis C (HCV). Subjects who are HBsAg negative and HBcAb positive are excluded unless negative for HBV DNA. Once past screening and enrolled into study, requirements for monitoring and antiviral treatment are enacted.
Subjects with a positive HCV antibody test should have HCV RNA levels measured. Subjects with positive (detectable) HCV RNA should be excluded.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
19/12/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
24/04/2026
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Actual
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Sample size
Target
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Accrual to date
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Final
107
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Clayton
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Recruitment postcode(s) [1]
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3168 - Clayton
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Recruitment outside Australia
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Argentina
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Buenos Aires
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China
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Guangdong
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China
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Jiangsu
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China
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Shanghai
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Czechia
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Praha 2
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France
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State/province [6]
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Pessac Cedex
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Germany
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Berlin
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Germany
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Freiburg
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Hungary
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Budapest
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Hungary
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Debrecen
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Israel
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Ramat Gan
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Japan
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Aichi
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Japan
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Tokyo
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Korea, Republic of
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Gwangju
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Poland
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Bydgoszcz
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Poland
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Poznan
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Poland
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Warszawa
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Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Russian Federation
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Yekaterinburg
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Spain
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Catalunya
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Spain
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Barcelona
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Taiwan
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Taiwan ROC
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Taiwan
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Taichung
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Thailand
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State/province [25]
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Bangkok
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Novartis Pharmaceuticals
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This study is designed to evaluate the safety, tolerability, pharmacokinetics and therapeutic efficacy of treatment with either VAY736 (ianalumab) or CFZ533 (iscalimab) in patients with systemic lupus erythematosus (SLE) to enable further development of these compounds as treatment in this disease population
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Trial website
https://clinicaltrials.gov/study/NCT03656562
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/study/NCT03656562
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