Study of Safety and Efficacy of Multiple Doses of CFZ533... | NCT03905525 | Trialant
NCT03905525
Sponsor
Novartis Pharmaceuticals
Status
Completed
Last Update Posted
May 18, 2026Actual
Enrollment
273Actual
Phase
Phase 2
Conditions
Sjögren Syndrome
Interventions
CFZ533
Placebo
Countries
United States
Argentina
Australia
Austria
Brazil
Canada
Chile
Colombia
France
Germany
Greece
Hungary
Israel
Italy
Japan
Netherlands
Portugal
Romania
Russia
South Korea
Sweden
Turkey (Türkiye)
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT03905525
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
CCFZ533B2201
Secondary IDs
ID
Type
Description
Link
2018-004476-35
EudraCT Number
Brief Title
Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome
Official Title
A 48-week, 6-arm, Randomized, Double-blind, Placebo-controlled Multicenter Trial to Assess the Safety and Efficacy of Multiple CFZ533 Doses Administered Subcutaneously in Two Distinct Populations of Patients With Sjogren's Syndrome (TWINSS)
Acronym
TWINSS
Organization
NovartisINDUSTRY
Status Module
Record Verification Date
Apr 2026
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Oct 1, 2019Actual
Primary Completion Date
Sep 28, 2022Actual
Completion Date
Jun 6, 2023Actual
First Submitted Date
Mar 25, 2019
First Submission Date that Met QC Criteria
Apr 4, 2019
First Posted Date
Apr 5, 2019Actual
Results Waived
Not provided
Results First Submitted Date
Jun 3, 2024
Results First Submitted that Met QC Criteria
Apr 23, 2026
Results First Posted Date
May 18, 2026Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Oct 4, 2022
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
May 18, 2026Actual
Last Update Submitted Date
Apr 23, 2026
Last Update Posted Date
May 18, 2026Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Novartis PharmaceuticalsINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This study was to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses of CFZ533 (iscalimab) in patients with Sjögren's Syndrome (SjS).
Detailed Description
This study consisted of a 6-week screening, 2 treatment periods of 24 weeks each, and a follow-up period of 12 weeks. In Periods 1 and 2, thirteen (13) treatment administration visits were planned, including a weekly loading regimen (2 visits) at the start of each treatment period. One administration equaled to two subcutaneous (s.c.) injections.
This study included two distinct cohorts termed Cohort 1 and Cohort 2.
Cohort 1: subjects with moderate-to-severe systemic and symptomatic disease involvement.
Cohort 2: subjects with low systemic disease involvement but high symptom burden.
Conditions Module
Conditions
Sjögren Syndrome
Keywords
Sjögren Syndrome (SjS)
sicca syndrome
dryness
fatigue
autoimmune disease
European League Against Rheumatism (EULAR)
EULAR Sjögren syndrome disease activity index (ESSDAI)
EULAR Sjögren syndrome patient reported index (ESSPRI)
monoclonal antibody
anti-CD40
iscalimab (CFZ533)
TWINSS
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
273Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Cohort 1 / Arm A
Experimental
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Drug: CFZ533
Cohort 1 / Arm B
Experimental
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Drug: CFZ533
Cohort 1 / Arm C
Experimental
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Drug: CFZ533
Cohort 1 / Arm D (Period 1)
Placebo Comparator
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
Other: Placebo
Cohort 1 / Arm D1 (Period 2)
Interventions
Name
Type
Description
Arm Group Labels
Other Names
CFZ533
Drug
Biological
Cohort 1 / Arm A
Cohort 1 / Arm B
Cohort 1 / Arm C
Cohort 1 / Arm D1 (Period 2)
Cohort 2 / Arm E
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Cohort 1: Change in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) Score From Baseline at 24 Weeks as Compared to Placebo
ESSDAI is a validated disease outcome measure for SjS that contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity. These scores are then summed across the 12 domains in a weighted manner to provide the total score. The domains (weights) are as follows: constitutional (3), lymphadenopathy (4), glandular (2), articular (2), cutaneous (3), pulmonary (5), renal (5), muscular (6), peripheral nervous system (PNS) (5), central nervous system (CNS) (5), hematological (2) and biological (1). The total score may vary between 0-123. It is considered low activity an ESSDAI < 5; moderate activity 5-13, and high activity if ESSDAI is >= 14.
Baseline, Week 24
Cohort 2: Change in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) Score From Baseline at 24 Weeks as Compared to Placebo.
The ESSPRI is a self-evaluation index for measuring symptoms including pain, fatigue and dryness. Each symptom was measured with a single 0 (no symptoms) to 10 (severe symptoms) numerical scale and the final ESSPRI score is calculated by averaging these domains with a maximum severity score of 10.
Baseline, Week 24
Secondary Outcomes
Measure
Description
Time Frame
Cohort 1: Change From Baseline in ESSPRI at Week 24
The ESSPRI is a self-evaluation index for measuring symptoms including pain, fatigue and dryness. Each symptom was measured with a single 0 (no symptoms) to 10 (severe symptoms) numerical scale and the final ESSPRI score is calculated by averaging these domains with a maximum severity score of 10.
Baseline, Week 24
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Both cohorts must have met all the following criteria:
Signed informed consent must be obtained prior to participation in the study
Male or female patient >= 18 years of age
Classification of Sjögren's Syndrome according to ACR/EULAR 2016 criteria (Shiboski et al 2016)
Seropositive for anti-Ro/SSA antibodies
Stimulated whole salivary flow rate of >= 0.1 mL/min
Able to communicate well with the Investigator to understand and comply with the requirements of the study
Inclusion criteria specific for Cohort 1:
Screening ESSDAI value >= 5 within the following 8 organ domains: constitutional, lymphadenopathy, glandular, articular, cutaneous, renal, hematologic and biologic
Patients with involvement of one or more of the remaining 4 domains are eligible but scores of these domains will not contribute to the assessment for eligibility for Cohort 1
At selected sites participating in Cohort 2, patients who based on the above criterion 7, do not qualify for Cohort 1, should be further evaluated for Cohort 2
Screening ESSPRI score of >= 5
Inclusion criteria specific for Cohort 2:
Screening ESSDAI value < 5 within 8 domains scored for inclusion criterion #7 Cohort 1
Hypergammaglobulinemia defined by IgG greater than upper limit of normal (ULN) or lymphocytopenia (less than lower limit of normal (LLN)) or hypocomplementemia (low C3, or low C4 - when considered due to disease activity and not due to genetic factors)
Score of >= 30 on IDEEL symptom bother questionnaire at Screening
Exclusion Criteria:
Sjögren's Syndrome overlap syndromes where another autoimmune rheumatic disease constitutes the principal illness
Use of other investigational drugs within 5 half-lives of enrollment or within 30 days whichever is longer, or longer if required by local regulations
Prior treatment with any of the following within 6 months prior to randomization:
B-cell depletors (e.g. rituximab, ianalumab) unless CD19+ B cell count have returned to ≥ 50 cells/µL
Fisher BA, Mariette X, Papas A, Grader-Beck T, Bootsma H, Ng WF, van Daele PLA, Finzel S, Noaiseh G, Elgueta S, Hermann J, McCoy SS, Akpek E, Bookman A, Sopala M, Montecchi-Palmer M, Luo WL, Scheurer C, Hueber W; TWINSS study group. Safety and efficacy of subcutaneous iscalimab (CFZ533) in two distinct populations of patients with Sjogren's disease (TWINSS): week 24 results of a randomised, double-blind, placebo-controlled, phase 2b dose-ranging study. Lancet. 2024 Aug 10;404(10452):540-553. doi: 10.1016/S0140-6736(24)01211-X. Epub 2024 Jul 31.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
Plan to Share IPD
Yes
Description
Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
The study was conducted at 71 sites in 23 countries. Argentina (2), Australia (1), Austria (2), Brazil (3), Canada (3), Chile (5), Colombia (3), France (5), Germany (4), Greece (1), Hungary (3), Israel (3), Italy (3), Japan (5), Republic of Korea (1), Netherlands (2), Portugal (4), Romania (2), Russia (6), Sweden (1), Turkey (1), United Kingdom (3), United States (8).
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
FG001
Cohort 1/Arm C: CFZ533 150 mg
Periods
Title
Milestones
Reasons Not Completed
Period 1 (up to Week 24): Cohorts 1&2
Type
Comment
Milestone Data
STARTED
Randomized Set (RAN) consisted of all randomized patients. Patients were analyzed according to the treatment they were assigned to at randomization.
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Apr 27, 2021
Jun 3, 2024
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Slovenia
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Patients were screened and enrolled into one of the 2 study Cohorts:
Cohort 1: At baseline subjects were randomized in ratio 1:1:1:1 into one of three CFZ533 (iscalimab) arms (A, B or C) or to placebo (Arm D). After completion of 24 weeks of treatment (Period 1) placebo patients (Arm D) will be switched to active treatment (Arm D1) for the subsequent 24 weeks (Period 2).
Cohort 2: At baseline subjects were randomized in ratio 1:1 to iscalimab (Arm E) or to placebo (Arm F). After completion of 24 weeks of treatment (Period 1), placebo patients (Arm F) will be switched to iscalimab active treatment (Arm F1) for the subsequent 24 weeks (Period 2).
All patients treated with iscalimab (Arms A,B,C,and E) in Period 1 continued the same study treatment in Period 2.
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Quadruple
Masking Description
Patients, investigator staff, persons performing the assessments, were blinded to the identity of the treatment within each cohort from the time of randomization until end of the study visit (week 60)
Period 2: 3 weekly subcutaneous (s.c.) loading doses of 600 mg iscalimab on Week 24, 25 and 26. After Week 26 and up to Week 46 (last dose), iscalimab was administered bi-weekly at 600 mg.
Drug: CFZ533
Cohort 2 / Arm E
Experimental
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Drug: CFZ533
Cohort 2 / Arm F (Period 1)
Placebo Comparator
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
Other: Placebo
Cohort 2 / Arm F1 (Period 2)
Experimental
Period 2: 3 weekly subcutaneous (s.c.) loading doses of iscalimab: 600 mg on Week 24, and 300 mg on Week 25 and Week 26. After Week 26, iscalimab was administered s.c. bi-weekly at 300 mg.
Drug: CFZ533
Cohort 2 / Arm F1 (Period 2)
iscalimab
Placebo
Other
liquid placebo for injections
Cohort 1 / Arm D (Period 1)
Cohort 2 / Arm F (Period 1)
Cohort 1: Change From Baseline in Score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Questionnaire at Week 24
The FACIT F (Functional Assessment of Chronic Illness Therapy-Fatigue) is a validated 13 item patient reported outcome measure assessing fatigue and its impact on daily functioning over the past 7 days, with total scores ranging from 0 to 52, where higher scores indicate less fatigue.
Baseline, 24 weeks
Cohort 1: Change From Baseline in Physician Global Assessment (PhGA) at Week 24
Physician's global assessment (PhGA) of disease activity was performed using a Visual Analog Scale (VAS) - an unnumbered 100 mm horizontal line ranging from "no disease activity" (score 0) to "maximal disease activity" (score 100). The assessment of patient's condition on the day is made by placing a vertical mark across the line.
Baseline, 24 weeks
Cohort 2: Change From Baseline in Score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Questionnaire at Week 24
The FACIT F (Functional Assessment of Chronic Illness Therapy-Fatigue) is a validated 13 item patient reported outcome measure assessing fatigue and its impact on daily functioning over the past 7 days, with total scores ranging from 0 to 52, where higher scores indicate less fatigue.
Baseline, 24 weeks
Cohort 2: Change From Baseline in Physician Global Assessment (PhGA) at Week 24
Physician's global assessment (PhGA) of disease activity was performed using a Visual Analog Scale (VAS) - an unnumbered 100 mm horizontal line ranging from "no disease activity" (score 0) to "maximal disease activity" (score 100). The assessment of patient's condition on the day is made by placing a vertical mark across the line.
Baseline, 24 weeks
Cohort 2: Change From Baseline in ESSDAI at Week 24
ESSDAI is a validated disease outcome measure for SjS that contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity. These scores are then summed across the 12 domains in a weighted manner to provide the total score. The domains (weights) are as follows: constitutional (3), lymphadenopathy (4), glandular (2), articular (2), cutaneous (3), pulmonary (5), renal (5), muscular (6), peripheral nervous system (PNS) (5), central nervous system (CNS) (5), hematological (2) and biological (1). The final score may vary between 0-123. It is considered low activity an ESSDAI < 5; moderate activity 5-13, and high activity if ESSDAI is >= 14.
Baseline, week 24
Cohort 2: Proportion of Subjects With at Least 12 Points Improvement Measured by Score of Impact of Dry Eye on Everyday Life (IDEEL) Questionnaire Symptom Bother Module at Week 24.
The Impact of Dry Eye on Everyday Life (IDEEL) questionnaire is a comprehensive dry eye specific questionnaire to evaluate treatment satisfaction, symptom-related bother and impact on daily life in a population with dry eye. This study only utilized the Dry Eye Symptom-Bother module.
The Dry Eye Symptom-Bother module of IDEEL is composed of a single dimension (20 items). A 4-point Likert-like scale is used: from "not at all" to "very much". Patients could also answer "I did not have this symptom / Not applicable". One item is scored on a 5-point Likert-like scale from "none of the time" to "all of the time". The range for the symptom-bother score is 0 to 100, with higher scores indicating greater symptom bother.
Baseline, Week 24
Cohort 1: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) up to Week 24
The distribution of adverse events in Treatment Period 1 was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) up to Week 24 (Period 1) is presented by actual treatment during Period 1, with data from separate cohort for the CFZ533 600mg and for the Placebo groups: CFZ533 600 mg, CFZ533 300 mg, CFZ533 150 mg and placebo.
Up to Week 24
Cohort 1: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) in All Study Periods
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) for period 2/3 or overall study is presented by actual treatment sequence during periods 1 and 2, where the CFZ533 600 mg - CFZ533 600 mg sequence included data from patients in separate cohort. CFZ533 600 mg 24 Weeks arm includes only patients from Placebo - CFZ533 600 mg arm, who took at least one CFZ533 600 mg dose in Period 2 (Patients who received Placebo in Period 1 and discontinued before Week 24 are not included). CFZ533 600 mg 48 Weeks arm includes all subjects from CFZ533 600 mg - CFZ533 600 mg arm, and subjects from CFZ533 150 mg - CFZ533 150 mg and CFZ533 300 mg - CFZ533 300 mg arms but only took the first or the first two loading dose(s) in period 1.
up to 14 weeks following the last dose of study treatment, up to maximum Week 60
Cohort 2: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) up to Week 24
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) up to Week 24 (Period 1) is presented by actual treatment during Period 1, with data from separate cohort for the CFZ533 600mg and for the Placebo groups: CFZ533 600 mg and placebo.
Up to Week 24
Cohort 2: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) in All Study Periods
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) for period 2/3 or overall study is presented by actual treatment sequence during periods 1 and 2, where the CFZ533 600 mg - CFZ533 600 mg sequence included data from patients in separate cohort. CFZ533 300 mg 24 Weeks includes only patients from Placebo - CFZ533 300 mg arm, who received Placebo in Period 1, and either took CFZ533 600 mg loading dose + at least two CFZ533 300 mg subsequent doses in Period 2 or missed CFZ533 600 mg loading dose and took at least one CFZ533 300 mg dose in Period 2 (Patients who received Placebo in Period 1 and discontinued before Week24 are not included). CFZ533 600 mg 48 Weeks arm includes all subjects from CFZ533 600 mg - CFZ533 600 mg arm.
up to 14 weeks following the last dose of study treatment, up to maximum Week 60
Cohort 1: Change From Baseline in Serum Free Light Kappa (FLCκ) Chains Levels
Serum samples for free light kappa (FLCκ) chains were collected and analyzed.
Cohort 1: Change From Baseline in Plasma CXCL-13 Levels
Plasma samples for Chemokine (C-X-C motif) ligand 13 (CXCL13), also known as B lymphocyte chemoattractant (BLC) or B cell-attracting chemokine 1 (BCA-1) were collected and analyzed.
Cohort 2: Change From Baseline in Plasma CXCL-13 Levels
Plasma samples for Chemokine (C-X-C motif) ligand 13 (CXCL13), also known as B lymphocyte chemoattractant (BLC) or B cell-attracting chemokine 1 (BCA-1) were collected and analyzed.
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
FG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
FG003
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
3 weekly subcutaneous (s.c.) loading doses of 600 mg iscalimab on Week 24, 25 and 26. After Week 26 and up to Week 46 (last dose), iscalimab was administered bi-weekly at 600 mg.
FG005
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
FG006
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
FG007
Cohort 2 / Arm F1 (Period 2): CFZ533 300mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab: 600 mg on Week 24, and 300 mg on Week 25 and Week 26. After Week 26, iscalimab was administered s.c. bi-weekly at 300 mg.
FG00043 subjects
FG00144 subjects
FG00243 subjects
FG00343 subjects
FG0040 subjects
FG00550 subjects
FG00650 subjects
FG0070 subjects
Full Analysis Set (FAS)
All patients to whom study treatment has been assigned by randomization. Patients were analyzed according to the treatment they have been assigned to during the randomization and the strata they actually belong to in case of misallocation of strata during the randomization process.
FG00043 subjects
FG00144 subjects
FG00243 subjects
FG00343 subjects
FG0040 subjects
FG00550 subjects
FG00650 subjects
FG0070 subjects
Safety Set (SAF)
All patients who received at least one dose of study treatment. Patients were analyzed according to the actual treatment received.
FG00043 subjects
FG00144 subjects
FG00243 subjectsone participant from Arm B only received the loading dose, and thus, was analyzed as part of Arm A for Safety Analyses
FG00343 subjects
FG0040 subjects
FG00550 subjects
FG00650 subjects
FG0070 subjects
Continued to Treatment Period 2
FG00041 subjects
FG00142 subjects
FG00241 subjects
FG00339 subjects
FG0040 subjects
FG00545 subjects
FG00648 subjects
FG0070 subjects
COMPLETED
Completed study treatment in Period 1
FG00041 subjects
FG00142 subjects
FG00241 subjects
FG00339 subjects
FG0040 subjects
FG00544 subjects
FG00648 subjects
FG0070 subjects
NOT COMPLETED
FG0002 subjects
FG0012 subjects
FG0022 subjects
FG0034 subjects
FG0040 subjects
FG0056 subjects
FG0062 subjects
FG0070 subjects
Type
Comment
Reasons
Adverse Event
FG0001 subjects
FG0011 subjects
FG0022 subjects
FG0034 subjects
FG0040 subjects
FG0053 subjects
FG0061 subjects
FG0070 subjects
Lost to Follow-up
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Physician Decision
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0030 subjects
FG004
Subject decision
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Period 2 (up to Week 48): Cohorts 1&2
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG00142 subjects
FG00241 subjects
FG00339 subjects
FG00441 subjects
FG0050 subjects
FG00648 subjects
FG00745 subjects
Continued to Post-Treatment Follow-up Period
FG0000 subjects
FG00142 subjects
FG00240 subjects
FG00339 subjects
COMPLETED
Completed study treatment in Period 2
FG0000 subjects
FG00138 subjects
FG00236 subjects
FG00339 subjects
NOT COMPLETED
FG0000 subjects
FG0014 subjects
FG0025 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0011 subjects
FG0021 subjects
FG003
Demographics and Baseline Characteristics were assessed in Period 1.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
BG001
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
BG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
BG003
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
BG004
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
BG005
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
BG006
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00043
BG00144
BG00243
BG00343
BG00450
BG00550
BG006273
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
Years
Title
Denominators
Categories
Title
Measurements
BG00053.3± 9.55
BG00149.3± 14.41
BG00248.7± 12.78
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00041
BG00142
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0011
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Cohort 1: Change in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) Score From Baseline at 24 Weeks as Compared to Placebo
ESSDAI is a validated disease outcome measure for SjS that contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity. These scores are then summed across the 12 domains in a weighted manner to provide the total score. The domains (weights) are as follows: constitutional (3), lymphadenopathy (4), glandular (2), articular (2), cutaneous (3), pulmonary (5), renal (5), muscular (6), peripheral nervous system (PNS) (5), central nervous system (CNS) (5), hematological (2) and biological (1). The total score may vary between 0-123. It is considered low activity an ESSDAI < 5; moderate activity 5-13, and high activity if ESSDAI is >= 14.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, Week 24
ID
Title
Description
OG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
OG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
OG003
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
OG00041
OG00142
OG00241
OG003
Title
Denominators
Categories
Title
Measurements
OG000-4.0± 0.73
OG001-7.0± 0.70
OG002-5.4± 0.71
OG003
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
ESSDAI score from baseline at 24 weeks as compared to placebo - Cohort 1
Mixed Models Analysis
0.0025
LS Mean difference CFZ533-Placebo
-3.0
95
-4.9
-1.1
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
Primary
Cohort 2: Change in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) Score From Baseline at 24 Weeks as Compared to Placebo.
The ESSPRI is a self-evaluation index for measuring symptoms including pain, fatigue and dryness. Each symptom was measured with a single 0 (no symptoms) to 10 (severe symptoms) numerical scale and the final ESSPRI score is calculated by averaging these domains with a maximum severity score of 10.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, Week 24
ID
Title
Description
OG000
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
Secondary
Cohort 1: Change From Baseline in ESSPRI at Week 24
The ESSPRI is a self-evaluation index for measuring symptoms including pain, fatigue and dryness. Each symptom was measured with a single 0 (no symptoms) to 10 (severe symptoms) numerical scale and the final ESSPRI score is calculated by averaging these domains with a maximum severity score of 10.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, Week 24
ID
Title
Description
OG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
OG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 1: Change From Baseline in Score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Questionnaire at Week 24
The FACIT F (Functional Assessment of Chronic Illness Therapy-Fatigue) is a validated 13 item patient reported outcome measure assessing fatigue and its impact on daily functioning over the past 7 days, with total scores ranging from 0 to 52, where higher scores indicate less fatigue.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, 24 weeks
ID
Title
Description
OG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
OG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 1: Change From Baseline in Physician Global Assessment (PhGA) at Week 24
Physician's global assessment (PhGA) of disease activity was performed using a Visual Analog Scale (VAS) - an unnumbered 100 mm horizontal line ranging from "no disease activity" (score 0) to "maximal disease activity" (score 100). The assessment of patient's condition on the day is made by placing a vertical mark across the line.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, 24 weeks
ID
Title
Description
OG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
OG002
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 2: Change From Baseline in Score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Questionnaire at Week 24
The FACIT F (Functional Assessment of Chronic Illness Therapy-Fatigue) is a validated 13 item patient reported outcome measure assessing fatigue and its impact on daily functioning over the past 7 days, with total scores ranging from 0 to 52, where higher scores indicate less fatigue.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, 24 weeks
ID
Title
Description
OG000
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
Secondary
Cohort 2: Change From Baseline in Physician Global Assessment (PhGA) at Week 24
Physician's global assessment (PhGA) of disease activity was performed using a Visual Analog Scale (VAS) - an unnumbered 100 mm horizontal line ranging from "no disease activity" (score 0) to "maximal disease activity" (score 100). The assessment of patient's condition on the day is made by placing a vertical mark across the line.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, 24 weeks
ID
Title
Description
OG000
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
Secondary
Cohort 2: Change From Baseline in ESSDAI at Week 24
ESSDAI is a validated disease outcome measure for SjS that contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity. These scores are then summed across the 12 domains in a weighted manner to provide the total score. The domains (weights) are as follows: constitutional (3), lymphadenopathy (4), glandular (2), articular (2), cutaneous (3), pulmonary (5), renal (5), muscular (6), peripheral nervous system (PNS) (5), central nervous system (CNS) (5), hematological (2) and biological (1). The final score may vary between 0-123. It is considered low activity an ESSDAI < 5; moderate activity 5-13, and high activity if ESSDAI is >= 14.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Posted
Least Squares Mean
Standard Error
Unit on a scale
Baseline, week 24
ID
Title
Description
OG000
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 2: Proportion of Subjects With at Least 12 Points Improvement Measured by Score of Impact of Dry Eye on Everyday Life (IDEEL) Questionnaire Symptom Bother Module at Week 24.
The Impact of Dry Eye on Everyday Life (IDEEL) questionnaire is a comprehensive dry eye specific questionnaire to evaluate treatment satisfaction, symptom-related bother and impact on daily life in a population with dry eye. This study only utilized the Dry Eye Symptom-Bother module.
The Dry Eye Symptom-Bother module of IDEEL is composed of a single dimension (20 items). A 4-point Likert-like scale is used: from "not at all" to "very much". Patients could also answer "I did not have this symptom / Not applicable". One item is scored on a 5-point Likert-like scale from "none of the time" to "all of the time". The range for the symptom-bother score is 0 to 100, with higher scores indicating greater symptom bother.
Full Analysis Set.
Posted
Count of Participants
Participants
Baseline, Week 24
ID
Title
Description
OG000
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
OG001
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 1: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) up to Week 24
The distribution of adverse events in Treatment Period 1 was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) up to Week 24 (Period 1) is presented by actual treatment during Period 1, with data from separate cohort for the CFZ533 600mg and for the Placebo groups: CFZ533 600 mg, CFZ533 300 mg, CFZ533 150 mg and placebo.
Safety Set (SAF).
Posted
Count of Participants
Participants
Up to Week 24
ID
Title
Description
OG000
Cohort 1: Placebo
Cohort 1: Placebo
OG001
Cohort 1: CFZ533 150 mg
Cohort 1: CFZ533 150 mg
OG002
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Secondary
Cohort 1: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) in All Study Periods
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) for period 2/3 or overall study is presented by actual treatment sequence during periods 1 and 2, where the CFZ533 600 mg - CFZ533 600 mg sequence included data from patients in separate cohort. CFZ533 600 mg 24 Weeks arm includes only patients from Placebo - CFZ533 600 mg arm, who took at least one CFZ533 600 mg dose in Period 2 (Patients who received Placebo in Period 1 and discontinued before Week 24 are not included). CFZ533 600 mg 48 Weeks arm includes all subjects from CFZ533 600 mg - CFZ533 600 mg arm, and subjects from CFZ533 150 mg - CFZ533 150 mg and CFZ533 300 mg - CFZ533 300 mg arms but only took the first or the first two loading dose(s) in period 1.
Safety Set (SAF).
Posted
Count of Participants
Participants
up to 14 weeks following the last dose of study treatment, up to maximum Week 60
Cohort 1/Arm D1: CFZ533 600 mg in Treatment Period 2 (Weeks 25-48)
OG001
Cohort 1/Arm C: CFZ533 150 mg (48 Weeks)
Cohort 1/Arm C: CFZ533 150 mg in Treatment Periods 1 and 2
Secondary
Cohort 2: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) up to Week 24
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) up to Week 24 (Period 1) is presented by actual treatment during Period 1, with data from separate cohort for the CFZ533 600mg and for the Placebo groups: CFZ533 600 mg and placebo.
Safety Set (SAF).
Posted
Count of Participants
Participants
Up to Week 24
ID
Title
Description
OG000
Cohort 2: Placebo
Cohort 2: Placebo
OG001
Cohort 2: CFZ533 600 mg
Cohort 2: CFZ533 600 mg
Units
Counts
Participants
OG000
Secondary
Cohort 2: Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs) in All Study Periods
The distribution of adverse events was done via the analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event (TESAEs) and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters.
Analyses of data in the Safety Set (SAF) for period 2/3 or overall study is presented by actual treatment sequence during periods 1 and 2, where the CFZ533 600 mg - CFZ533 600 mg sequence included data from patients in separate cohort. CFZ533 300 mg 24 Weeks includes only patients from Placebo - CFZ533 300 mg arm, who received Placebo in Period 1, and either took CFZ533 600 mg loading dose + at least two CFZ533 300 mg subsequent doses in Period 2 or missed CFZ533 600 mg loading dose and took at least one CFZ533 300 mg dose in Period 2 (Patients who received Placebo in Period 1 and discontinued before Week24 are not included). CFZ533 600 mg 48 Weeks arm includes all subjects from CFZ533 600 mg - CFZ533 600 mg arm.
Safety Set (SAF).
Posted
Count of Participants
Participants
up to 14 weeks following the last dose of study treatment, up to maximum Week 60
ID
Title
Description
OG000
Cohort 2 / Arm F1 (Period 2): CFZ533 300mg (From Week 24)
Cohort 2 / Arm F1: CFZ533 300 mg in Treatment Period 2 (Weeks 25-48)
OG001
Cohort 2/Arm E: CFZ533 600 mg (48 Weeks)
Cohort 2/Arm E: CFZ533 600 mg in Treatment Periods 1 and 2
Secondary
Cohort 1: Change From Baseline in Serum Free Light Kappa (FLCκ) Chains Levels
Serum samples for free light kappa (FLCκ) chains were collected and analyzed.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Cohort 1: In Arm D (Period 1), placebo treatment is administered up to Week 22 (last dose administration visit in Period 1). In Arm D1 (Period 2), CFZ533 600 mg is administered from Week 25 up to Week 46 (last dose administration visit in Period 2).
OG001
Cohort 1/Arm C: CFZ533 150 mg
Cohort 1/Arm C: CFZ533 150 mg in Treatment Periods 1 and 2
OG002
Cohort 1/Arm B: CFZ533 300 mg
Cohort 1/Arm B: CFZ533 300 mg in Treatment Periods 1 and 2
OG003
Cohort 1/Arm A: CFZ533 600 mg
Secondary
Cohort 2: Change From Baseline in Serum Free Light Kappa (FLCκ) Chains Levels
Serum samples for free light kappa (FLCκ) chains were collected and analyzed.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Cohort 1: In Arm D (Period 1), placebo treatment is administered up to Week 22 (last dose administration visit in Period 1). In Arm D1 (Period 2), CFZ533 600 mg is administered from Week 25 up to Week 46 (last dose administration visit in Period 2).
OG001
Cohort 1/Arm C: CFZ533 150 mg
Cohort 1/Arm C: CFZ533 150 mg in Treatment Periods 1 and 2
OG002
Cohort 1/Arm B: CFZ533 300 mg
Cohort 1/Arm B: CFZ533 300 mg in Treatment Periods 1 and 2
OG003
Cohort 1/Arm A: CFZ533 600 mg
Secondary
Cohort 2: Change From Baseline in Immunoglobulin G (IgG) Levels
Plasma samples for Immunoglobulin G (IgG) were collected and analyzed.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Cohort 1: In Arm D (Period 1), placebo treatment is administered up to Week 22 (last dose administration visit in Period 1). In Arm D1 (Period 2), CFZ533 600 mg is administered from Week 25 up to Week 46 (last dose administration visit in Period 2).
OG001
Cohort 1/Arm C: CFZ533 150 mg
Cohort 1/Arm C: CFZ533 150 mg in Treatment Periods 1 and 2
OG002
Cohort 1/Arm B: CFZ533 300 mg
Cohort 1/Arm B: CFZ533 300 mg in Treatment Periods 1 and 2
OG003
Cohort 1/Arm A: CFZ533 600 mg
Secondary
Cohort 2: Change From Baseline in Immunoglobulin M (IgM) Levels
Plasma samples for Immunoglobulin M (IgM) were collected and analyzed.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
Cohort 1: Change From Baseline in Plasma CXCL-13 Levels
Plasma samples for Chemokine (C-X-C motif) ligand 13 (CXCL13), also known as B lymphocyte chemoattractant (BLC) or B cell-attracting chemokine 1 (BCA-1) were collected and analyzed.
Full Analysis Set. Only participants in cohort 1 with a value at both Baseline and post-baseline visit included.
Cohort 1: In Arm D (Period 1), placebo treatment is administered up to Week 22 (last dose administration visit in Period 1). In Arm D1 (Period 2), CFZ533 600 mg is administered from Week 25 up to Week 46 (last dose administration visit in Period 2).
OG001
Cohort 1/Arm C: CFZ533 150 mg
Cohort 1/Arm C: CFZ533 150 mg in Treatment Periods 1 and 2
OG002
Cohort 1/Arm B: CFZ533 300 mg
Cohort 1/Arm B: CFZ533 300 mg in Treatment Periods 1 and 2
OG003
Secondary
Cohort 2: Change From Baseline in Plasma CXCL-13 Levels
Plasma samples for Chemokine (C-X-C motif) ligand 13 (CXCL13), also known as B lymphocyte chemoattractant (BLC) or B cell-attracting chemokine 1 (BCA-1) were collected and analyzed.
Full Analysis Set. Only participants in cohort 2 with a value at both Baseline and post-baseline visit included.
On-treatment adverse events and deaths were reported from first dose of study treatment to 14 weeks after last dose of study medication, up to Week 60.
Description
Any sign or symptom that occurred during the conduct of the trial and safety follow-up. The safety analysis were done on the safety population, which included all randomized subjects who received at least one dose of study medication. Patients were analyzed according to the actual treatment received.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Cohort 1 / Arm D (Period 1): Placebo
Placebo treatment is administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
3 weekly subcutaneous (s.c.) loading doses of 600 mg iscalimab on Week 24, 25 and 26. After Week 26 and up to Week 46 (last dose), iscalimab was administered bi-weekly at 600 mg.
0
41
4
41
28
41
EG002
Cohort 1/Arm C: CFZ533 150 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 150 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 150 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
0
44
6
44
33
44
EG003
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
1
42
6
42
34
42
EG004
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
0
44
6
44
34
44
EG005
Cohort 2/Arm F: Placebo
Placebo treatment was administered subcutaneous (s.c.) weekly for the first 3 doses, then bi-weekly from Week 2 to Week 22 in Period 1.
0
50
2
50
27
50
EG006
Cohort 2 / Arm F1 (Period 2): CFZ533 300mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab: 600 mg on Week 24, and 300 mg on Week 25 and Week 26. After Week 26, iscalimab was administered s.c. bi-weekly at 300 mg.
0
44
5
44
32
44
EG007
Cohort 2/Arm E: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
1
50
6
50
41
50
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG0030 affected42 at risk
EG0041 affected44 at risk
EG0050 affected50 at risk
EG0060 affected44 at risk
EG0070 affected50 at risk
Angina pectoris
Cardiac disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Arteriosclerosis coronary artery
Cardiac disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Cardiac failure congestive
Cardiac disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Angle closure glaucoma
Eye disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Enteritis
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Haematochezia
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Pancreatitis acute
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Salivary gland cyst
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Pyrexia
General disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Cholecystitis
Hepatobiliary disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Appendicitis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
COVID-19
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Laryngitis bacterial
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Lower respiratory tract infection
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Meningitis aseptic
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Pneumocystis jirovecii pneumonia
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Postoperative wound infection
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Respiratory tract infection viral
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Retroperitoneal abscess
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Tuberculosis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Wound abscess
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Ankle fracture
Injury, poisoning and procedural complications
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Fibula fracture
Injury, poisoning and procedural complications
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Tibia fracture
Injury, poisoning and procedural complications
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Hand deformity
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Osteoarthritis
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Sjogren's syndrome
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Bladder cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Breast cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Cerebrovascular disorder
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Migraine
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Transient ischaemic attack
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Glomerulonephritis
Renal and urinary disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Nephrolithiasis
Renal and urinary disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Renal colic
Renal and urinary disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Pelvic organ prolapse
Reproductive system and breast disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Pulmonary oedema
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Respiratory distress
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Iron deficiency anaemia
Blood and lymphatic system disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0021 affected44 at risk
EG0030 affected42 at risk
EG004
Leukopenia
Blood and lymphatic system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0021 affected44 at risk
EG003
Thyroid mass
Endocrine disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0022 affected44 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Parotid gland enlargement
Gastrointestinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Asthenia
General disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0021 affected44 at risk
EG003
Fatigue
General disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0025 affected44 at risk
EG003
Pyrexia
General disorders
MedDRA (26.0)
Systematic Assessment
EG0003 affected43 at risk
EG0012 affected41 at risk
EG0026 affected44 at risk
EG003
COVID-19
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG00110 affected41 at risk
EG00210 affected44 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Herpes simplex
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0010 affected41 at risk
EG0024 affected44 at risk
EG003
Influenza
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0013 affected41 at risk
EG0022 affected44 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG0016 affected41 at risk
EG0025 affected44 at risk
EG003
Oral herpes
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG0010 affected41 at risk
EG0023 affected44 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Rhinitis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Sinusitis
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0021 affected44 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0014 affected41 at risk
EG0023 affected44 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG0013 affected41 at risk
EG0023 affected44 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0010 affected41 at risk
EG0023 affected44 at risk
EG003
Immunisation reaction
Injury, poisoning and procedural complications
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0013 affected41 at risk
EG0021 affected44 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0004 affected43 at risk
EG0012 affected41 at risk
EG0026 affected44 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0012 affected41 at risk
EG0024 affected44 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0021 affected44 at risk
EG003
Neck pain
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0002 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Headache
Nervous system disorders
MedDRA (26.0)
Systematic Assessment
EG0005 affected43 at risk
EG0011 affected41 at risk
EG0029 affected44 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0011 affected41 at risk
EG0023 affected44 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Alopecia
Skin and subcutaneous tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0011 affected41 at risk
EG0020 affected44 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0001 affected43 at risk
EG0010 affected41 at risk
EG0021 affected44 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0012 affected41 at risk
EG0021 affected44 at risk
EG003
Haematoma
Vascular disorders
MedDRA (26.0)
Systematic Assessment
EG0000 affected43 at risk
EG0010 affected41 at risk
EG0020 affected44 at risk
EG003
Hypertension
Vascular disorders
MedDRA (26.0)
Systematic Assessment
EG0004 affected43 at risk
EG0010 affected41 at risk
EG0022 affected44 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
ESSDAI score from baseline at 24 weeks as compared to placebo - Cohort 1
Mixed Models Analysis
0.1578
LS Mean difference CFZ533-Placebo
-1.4
95
-3.3
0.5
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG003
ESSDAI score from baseline at 24 weeks as compared to placebo - Cohort 1
Mixed Models Analysis
0.0037
LS Mean difference CFZ533-Placebo
-2.9
2-Sided
95
-4.9
-1.0
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG00045
OG00149
Title
Denominators
Categories
Title
Measurements
OG000-1.21± 0.271
OG001-1.79± 0.258
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
ESSPRI score from baseline at 24 weeks as compared to placebo - Cohort 2
Mixed Models Analysis
0.1210
LS Mean difference CFZ533-Placebo
-0.57
2-Sided
95
-1.30
0.15
MMRM that includes treatment, visit, treatment by visit interaction and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG003
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
OG00039
OG00142
OG00240
OG00339
Title
Denominators
Categories
Title
Measurements
OG000-1.3± 0.31
OG001-1.8± 0.30
OG002-1.6± 0.31
OG003-1.8± 0.31
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
ESSPRI at Week 24 - Cohort 1
Mixed Models Analysis
0.2078
LS Mean difference CFZ533-Placebo
-0.5
2-Sided
95
-1.4
0.3
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG002
ESSPRI at Week 24 - Cohort 1
Mixed Models Analysis
0.5132
LS Mean difference CFZ533-Placebo
-0.3
2-Sided
95
-1.1
0.6
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG003
ESSPRI at Week 24 - Cohort 1
Mixed Models Analysis
0.1998
LS Mean difference CFZ533-Placebo
-0.5
2-Sided
0.1998
-1.4
0.3
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG003
Cohort 1/Arm A: CFZ533 600 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Weeks 0, 1 and 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 600 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
OG00040
OG00142
OG00240
OG00339
Title
Denominators
Categories
Title
Measurements
OG0007.0± 1.48
OG0018.6± 1.45
OG0028.0± 1.47
OG00310.3± 1.50
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
FACIT-F at Week 24 - Cohort 1
Mixed Models Analysis
0.4363
LS Mean difference CFZ533-Placebo
1.6
2-Sided
95
-2.4
5.6
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG002
FACIT-F at Week 24 - Cohort 1
Mixed Models Analysis
0.6181
LS Mean difference CFZ533-Placebo
1.0
2-Sided
95
-3.0
5.0
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG003
FACIT-F at Week 24 - Cohort 1
Mixed Models Analysis
0.1050
LS Mean difference CFZ533-Placebo
3.3
2-Sided
95
-0.7
7.3
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG003
Cohort 1/Arm B: CFZ533 300 mg
3 weekly subcutaneous (s.c.) loading doses of iscalimab were 600 mg on Week 0, and 300 mg on Week 1 and Week 2. From Week 2 and up to Week 46 (last dose), iscalimab was administered s.c. bi-weekly at 300 mg. To maintain blinding in Period 2, placebo was administered at Week 25.
Units
Counts
Participants
OG00036
OG00140
OG00239
OG00335
Title
Denominators
Categories
Title
Measurements
OG000-23.9± 2.94
OG001-31.6± 2.83
OG002-27.0± 2.87
OG003-30.8± 3.00
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
PhGA at Week 24 - Cohort 1
Mixed Models Analysis
0.0561
LS Mean difference CFZ533-Placebo
-7.7
2-Sided
95
-15.6
0.2
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG002
PhGA at Week 24 - Cohort 1
Mixed Models Analysis
0.4433
LS Mean difference CFZ533-Placebo
-3.1
2-Sided
95
-11.0
4.8
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG000
OG003
PhGA at Week 24 - Cohort 1
Mixed Models Analysis
0.0974
LS Mean difference CFZ533-Placebo
-6.9
2-Sided
95
-15.0
1.3
MMRM that includes treatment, visit, treatment by visit interaction, stratification factor baseline ESSDAI score (< 10 or >= 10 based on weighted scores), and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG00045
OG00149
Title
Denominators
Categories
Title
Measurements
OG0005.7± 1.32
OG0017.3± 1.25
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
FACIT-F at Week 24 - Cohort 2
Mixed Models Analysis
0.3675
LS Mean difference CFZ533-Placebo
1.6
2-Sided
95
-1.9
5.1
MMRM that includes treatment, visit, treatment by visit interaction and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
OG00045
OG00146
Title
Denominators
Categories
Title
Measurements
OG000-10.4± 2.37
OG001-15.8± 2.29
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
PhGA at Week 24 - Cohort 2
Mixed Models Analysis
0.1014
LS Mean difference CFZ533-Placebo
-5.4
2-Sided
95
-11.8
1.1
MMRM that includes treatment, visit, treatment by visit interaction and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
Units
Counts
Participants
OG00045
OG00147
Title
Denominators
Categories
Title
Measurements
OG0000.2± 0.33
OG001-0.3± 0.32
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
ESSDAI at Week 24 - Cohort 2
Mixed Models Analysis
0.2694
LS Mean difference CFZ533-Placebo
-0.5
2-Sided
95
-1.4
0.4
MMRM that includes treatment, visit, treatment by visit interaction and region as factors and baseline value of corresponding parameter as continuous covariates.
Other
Units
Counts
Participants
OG00050
OG00150
Title
Denominators
Categories
Title
Measurements
OG00020
OG00124
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
IDEEL using non-responder imputation up to Week 24 - Cohort 2 (Full Analysis Set)
Fisher Exact
0.5459
Clopper-Pearson method
8.0
2-Sided
95
-11.4
27.4
Difference CFZ533-Placebo
Other
OG003
Cohort 1: CFZ533 600 mg
Cohort 1: CFZ533 600 mg
Units
Counts
Participants
OG00043
OG00144
OG00242
OG00344
Title
Denominators
Categories
Death
Title
Measurements
OG0000
OG0010
OG0020
OG0030
Adverse Event
Title
Measurements
OG00031
OG00138
OG00232
OG003
Serious Adverse Event
Title
Measurements
OG0001
OG0011
OG0023
OG003
AE leading to study medication discontinuation
Title
Measurements
OG0001
OG0011
OG0021
OG003
OG002
Cohort 1/Arm B: CFZ533 300 mg (48 Weeks)
Cohort 1/Arm B: CFZ533 300 mg in Treatment Periods 1 and 2
OG003
Cohort 1/Arm A: CFZ533 600 mg (48 Weeks)
Cohort 1/Arm A: CFZ533 600 mg in Treatment Periods 1 and 2
OG004
Any CFZ533 600 mg
All participants from cohort 1 who received CFZ533 600 mg in all Study Periods (including placebo patients who switched to CFZ533 600 mg at Week 24)
OG005
Any CFZ533
All participants from cohort 1 who received a dose of CFZ533 during the study
Units
Counts
Participants
OG00041
OG00144
OG00242
OG00344
OG00485
OG005171
Title
Denominators
Categories
Death
Title
Measurements
OG0000
OG0010
OG0021
OG0030
OG0040
OG0051
Adverse Event
Title
Measurements
OG00034
OG00140
OG00238
OG003
Serious Adverse Event
Title
Measurements
OG0004
OG0016
OG0026
OG003
AE leading to study medication discontinuation
Title
Measurements
OG0000
OG0012
OG0023
OG003
50
OG00150
Title
Denominators
Categories
Death
Title
Measurements
OG0000
OG0010
Adverse Event
Title
Measurements
OG00032
OG00141
Serious Adverse Event
Title
Measurements
OG0002
OG0012
AE leading to study medication discontinuation
Title
Measurements
OG0003
OG0011
OG002
Any CFZ533
All participants from cohort 2 who received a dose of CFZ533 during the study
Units
Counts
Participants
OG00044
OG00150
OG00294
Title
Denominators
Categories
Death
Title
Measurements
OG0000
OG0011
OG0021
Adverse Event
Title
Measurements
OG00035
OG00144
OG00279
Serious Adverse Event
Title
Measurements
OG0005
OG0016
OG00211
AE leading to study medication discontinuation
Title
Measurements
OG0000
OG0013
OG0023
Cohort 1/Arm A: CFZ533 600 mg in Treatment Periods 1 and 2
Units
Counts
Participants
OG00042
OG00141
OG00241
OG00338
Title
Denominators
Categories
Week 4
ParticipantsOG00042
ParticipantsOG00140
ParticipantsOG00241
ParticipantsOG00337
Title
Measurements
OG000-1.9± 1.85
OG001-7.2± 1.86
OG002-5.4± 1.86
OG003
Week 12
ParticipantsOG00039
ParticipantsOG00141
ParticipantsOG00239
ParticipantsOG00338
Week 24 (End Treatment Period 1)
ParticipantsOG00040
ParticipantsOG00140
ParticipantsOG00239
ParticipantsOG00335
Week 32
ParticipantsOG00040
ParticipantsOG00139
ParticipantsOG00237
ParticipantsOG00333
Week 40
ParticipantsOG00038
ParticipantsOG00135
ParticipantsOG00236
ParticipantsOG00334
Week 48 (End Treatment Period 2)
ParticipantsOG00039
ParticipantsOG00136
ParticipantsOG00235
ParticipantsOG00336
FUP2 (Week 56)
ParticipantsOG00035
ParticipantsOG00137
ParticipantsOG00237
ParticipantsOG00334
FUP 3 (Week 60)
ParticipantsOG00036
ParticipantsOG00137
ParticipantsOG00236
ParticipantsOG00334
46
OG00149
Title
Denominators
Categories
Week 4
ParticipantsOG00045
ParticipantsOG00149
Title
Measurements
OG0000.3± 0.92
OG001-4.3± 0.87
Week 12
ParticipantsOG00046
ParticipantsOG00148
Title
Measurements
OG0000.1± 1.10
OG001
Week 24 (End Treatment Period 1)
ParticipantsOG00046
ParticipantsOG00148
Title
Measurements
OG000-0.2± 0.90
OG001
Week 32
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-6.0± 0.93
OG001
Week 40
ParticipantsOG00044
ParticipantsOG00141
Title
Measurements
OG000-7.8± 0.93
OG001
Week 48 (End Treatment Period 2)
ParticipantsOG00044
ParticipantsOG00142
Title
Measurements
OG000-9.3± 0.95
OG001
FUP2 (Week 56)
ParticipantsOG00045
ParticipantsOG00142
Title
Measurements
OG000-6.1± 1.00
OG001
FUP 3 (Week 60)
ParticipantsOG00044
ParticipantsOG00143
Title
Measurements
OG000-1.9± 1.11
OG001
Cohort 1/Arm A: CFZ533 600 mg in Treatment Periods 1 and 2
Units
Counts
Participants
OG00043
OG00143
OG00243
OG00341
Title
Denominators
Categories
Week 4
ParticipantsOG00043
ParticipantsOG00142
ParticipantsOG00243
ParticipantsOG00340
Title
Measurements
OG0000.1± 0.31
OG001-0.9± 0.31
OG002-0.6± 0.31
OG003
Week 8
ParticipantsOG00041
ParticipantsOG00141
ParticipantsOG00241
ParticipantsOG00341
Week 12
ParticipantsOG00040
ParticipantsOG00143
ParticipantsOG00240
ParticipantsOG00340
Week 16
ParticipantsOG00041
ParticipantsOG00142
ParticipantsOG00240
ParticipantsOG00340
Week 20
ParticipantsOG00041
ParticipantsOG00143
ParticipantsOG00241
ParticipantsOG00339
Week 24 (End Treatment Period 1)
ParticipantsOG00041
ParticipantsOG00142
ParticipantsOG00241
ParticipantsOG00339
Week 28
ParticipantsOG00040
ParticipantsOG00141
ParticipantsOG00241
ParticipantsOG00339
Week 32
ParticipantsOG00041
ParticipantsOG00139
ParticipantsOG00237
ParticipantsOG00335
Week 40
ParticipantsOG00039
ParticipantsOG00137
ParticipantsOG00238
ParticipantsOG00336
Week 48 (End Treatment Period 2)
ParticipantsOG00039
ParticipantsOG00138
ParticipantsOG00235
ParticipantsOG00338
FUP1 (Week 52)
ParticipantsOG00038
ParticipantsOG00139
ParticipantsOG00238
ParticipantsOG00341
FUP2 (Week 56)
ParticipantsOG00035
ParticipantsOG00138
ParticipantsOG00236
ParticipantsOG00336
FUP 3 (Week 60)
ParticipantsOG00039
ParticipantsOG00140
ParticipantsOG00237
ParticipantsOG00338
47
OG00149
Title
Denominators
Categories
Week 4
ParticipantsOG00045
ParticipantsOG00149
Title
Measurements
OG000-0.8± 0.25
OG001-0.8± 0.24
Week 8
ParticipantsOG00047
ParticipantsOG00147
Title
Measurements
OG000-0.5± 0.28
OG001
Week 12
ParticipantsOG00046
ParticipantsOG00148
Title
Measurements
OG000-0.4± 0.30
OG001
Week 16
ParticipantsOG00045
ParticipantsOG00146
Title
Measurements
OG000-0.6± 0.32
OG001
Week 20
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-0.7± 0.32
OG001
Week 24 (End Treatment Period 1)
ParticipantsOG00045
ParticipantsOG00149
Title
Measurements
OG0000.0± 0.42
OG001
Week 28
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-1.3± 0.38
OG001
Week 32
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-1.5± 0.35
OG001
Week 40
ParticipantsOG00043
ParticipantsOG00141
Title
Measurements
OG000-2.6± 0.43
OG001
Week 48 (End Treatment Period 2)
ParticipantsOG00043
ParticipantsOG00142
Title
Measurements
OG000-3.2± 0.43
OG001
FUP1 (Week 52)
ParticipantsOG00045
ParticipantsOG00141
Title
Measurements
OG000-4.0± 0.45
OG001
FUP2 (Week 56)
ParticipantsOG00044
ParticipantsOG00142
Title
Measurements
OG000-3.3± 0.46
OG001
FUP 3 (Week 60)
ParticipantsOG00045
ParticipantsOG00143
Title
Measurements
OG000-2.4± 0.46
OG001
Cohort 1/Arm A: CFZ533 600 mg in Treatment Periods 1 and 2
Units
Counts
Participants
OG00043
OG00143
OG00243
OG00341
Title
Denominators
Categories
Week 4
ParticipantsOG00043
ParticipantsOG00142
ParticipantsOG00243
ParticipantsOG00340
Title
Measurements
OG0000.0± 0.04
OG001-0.1± 0.04
OG002-0.1± 0.04
OG003
Week 8
ParticipantsOG00041
ParticipantsOG00142
ParticipantsOG00241
ParticipantsOG00341
Week 12
ParticipantsOG00040
ParticipantsOG00143
ParticipantsOG00240
ParticipantsOG00340
Week 16
ParticipantsOG00041
ParticipantsOG00142
ParticipantsOG00240
ParticipantsOG00340
Week 20
ParticipantsOG00041
ParticipantsOG00143
ParticipantsOG00241
ParticipantsOG00339
Week 24 (End Treatment Period 1)
ParticipantsOG00041
ParticipantsOG00142
ParticipantsOG00241
ParticipantsOG00339
Week 28
ParticipantsOG00040
ParticipantsOG00141
ParticipantsOG00241
ParticipantsOG00339
Week 32
ParticipantsOG00041
ParticipantsOG00139
ParticipantsOG00237
ParticipantsOG00335
Week 40
ParticipantsOG00039
ParticipantsOG00137
ParticipantsOG00238
ParticipantsOG00336
Week 48 (End Treatment Period 2)
ParticipantsOG00039
ParticipantsOG00138
ParticipantsOG00235
ParticipantsOG00338
FUP1 (Week 52)
ParticipantsOG00038
ParticipantsOG00139
ParticipantsOG00238
ParticipantsOG00341
FUP2 (Week 56)
ParticipantsOG00035
ParticipantsOG00138
ParticipantsOG00236
ParticipantsOG00336
FUP 3 (Week 60)
ParticipantsOG00039
ParticipantsOG00140
ParticipantsOG00237
ParticipantsOG00338
47
OG00149
Title
Denominators
Categories
Week 4
ParticipantsOG00045
ParticipantsOG00149
Title
Measurements
OG0000.0± 0.03
OG001-0.1± 0.03
Week 8
ParticipantsOG00047
ParticipantsOG00147
Title
Measurements
OG0000.0± 0.04
OG001
Week 12
ParticipantsOG00046
ParticipantsOG00148
Title
Measurements
OG0000.0± 0.05
OG001
Week 16
ParticipantsOG00045
ParticipantsOG00146
Title
Measurements
OG000-0.1± 0.06
OG001
Week 20
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-0.1± 0.06
OG001
Week 24 (End Treatment Period 1)
ParticipantsOG00045
ParticipantsOG00148
Title
Measurements
OG0000.0± 0.06
OG001
Week 28
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-0.2± 0.06
OG001
Week 32
ParticipantsOG00044
ParticipantsOG00147
Title
Measurements
OG000-0.2± 0.07
OG001
Week 40
ParticipantsOG00043
ParticipantsOG00141
Title
Measurements
OG000-0.3± 0.07
OG001
Week 48 (End Treatment Period 2)
ParticipantsOG00043
ParticipantsOG00142
Title
Measurements
OG000-0.3± 0.07
OG001
FUP1 (Week 52)
ParticipantsOG00045
ParticipantsOG00141
Title
Measurements
OG000-0.3± 0.06
OG001
FUP2 (Week 56)
ParticipantsOG00044
ParticipantsOG00142
Title
Measurements
OG000-0.2± 0.72
OG001
FUP 3 (Week 60)
ParticipantsOG00045
ParticipantsOG00143
Title
Measurements
OG000-0.1± 0.08
OG001
Cohort 1/Arm A: CFZ533 600 mg
Cohort 1/Arm A: CFZ533 600 mg in Treatment Periods 1 and 2