Primary Sjögren's SyndromePhase 2NCT02291029

Safety, Pharmacokinetics and Preliminary Efficacy Study of CFZ533 in Patients With Primary Sjögren's Syndrome

A Phase 2 study testing CFZ533 active - Cohort 1, CFZ533 active - Cohort 2 and CFZ533 active -Cohort 3 in people with Primary Sjögren's Syndrome, run by Novartis Pharmaceuticals at 9 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study did evaluate the safety,tolerability and preliminary therapeutic efficacy of multiple doses of intravenous infusion of CFZ533 monoclonal antibody in patients with primary Sjögren's syndrome(pSS) Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Primary Sjögren's Syndrome
What is being tested?: CFZ533 active - Cohort 1, CFZ533 active - Cohort 2 and CFZ533 active -Cohort 3
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 69 participants
Where: 9 sitesin 5 countries
Study dates: 22 Oct 2014to 29 Jun 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Diagnosis of primary Sjögren's syndrome
ESSDAI score ≥ 6

Who cannot join

Secondary Sjögren's syndrome
Receiving cyclosphosphamide, corticosteroid bolus with dose over 1 mg/kg, rituximab, belimunab, other immunosuppressives.
At significant risk for thromboembolic event
Clinically significant systemic infection
Significant elevated risk for infection

Trial locations and contacts

9 sites in 5 countries.

Country

City, postcode or hospital

9 sites in 5 countries

United States3 sites

Novartis Investigative Site
Boston, Massachusetts, 02111
Novartis Investigative Site
Duncansville, Pennsylvania, 16635
Novartis Investigative Site
Mineola, New York, 11501

Other trials in United States

United Kingdom3 sites

Novartis Investigative Site
Edgbaston, Birmingham, B15 2WB
Novartis Investigative Site
London, EC14 7BE
Novartis Investigative Site
Newcastle upon Tyne, NE2 4HH

Other trials in United Kingdom

Germany1 site

Novartis Investigative Site
Berlin, 10117

Other trials in Germany

Hungary1 site

Novartis Investigative Site
Debrecen, 4032

Other trials in Hungary

Switzerland1 site

Novartis Investigative Site
Basel, 4031

Other trials in Switzerland

About this study

Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CFZ533 in Patients With Primary Sjögren's Syndrome

Conditions: Primary Sjögren's Syndrome
Being tested: CFZ533 active - Cohort 1, CFZ533 placebo- Cohort 1, CFZ533 active - Cohort 2, CFZ533 placebo - Cohort 2, CFZ533 active -Cohort 3
Keywords: Sjögren's syndrome, CFZ533, ESSDAI
Medical subject headings: Sjogren's Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

CFZ533 active - Cohort 1Drug
multiple doses of CFZ533 s.c. injection
CFZ533 placebo- Cohort 1Drug
multiple doses of placebo s.c. injection
CFZ533 active - Cohort 2Drug
multiple doses of CFZ533 intravenous infusion
CFZ533 placebo - Cohort 2Drug
multiple doses of placebo intravenous infusion
CFZ533 active -Cohort 3Drug
multiple doses of CFZ533 s.c. injection
CFZ533 active - Cohort 3Drug
Single dose of CFZ533 i.v. infusion and multiple doses of CFZ533 s.c. injection

Study groups in NCT02291029
Study group	Role	What they receive
CFZ533 active- Cohort 2	Experimental	multiple doses of CFZ533 intravenous infusion Receives: Drug: CFZ533 active - Cohort 2
CFZ533 placebo- Cohort 2	Placebo comparator	multiple doses of placebo intravenous infusion Receives: Drug: CFZ533 placebo - Cohort 2
CFZ533 active - Cohort 1	Experimental	multiple doses of CFZ533 s.c. injection Receives: Drug: CFZ533 active - Cohort 1
CFZ533 placebo - Cohort 1	Placebo comparator	multiple doses of placebo s.c. injection Receives: Drug: CFZ533 placebo- Cohort 1
CFZ533 Treatment Arm 1 - Cohort 3	Experimental	multiple doses of CFZ533 s.c. injection Receives: Drug: CFZ533 active -Cohort 3
CFZ533 Treatment Arm 2 - Cohort 3	Experimental	Single dose of CFZ533 i.v. infusion and multiple doses of CFZ533 s.c. injection Receives: Drug: CFZ533 active - Cohort 3

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)
The effect of CFZ533 on clinical disease activity was measured by the change in ESSDAI (EULAR Sjögren's syndrome disease activity index) between baseline and week 12. The instrument 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 (range 0-123). A reduction from baseline indicates improvement in patients.
Measured: Baseline and Week 12

Secondary outcomes (6)

Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI)
The ESSPRI is a patient self-reported outcome measure to assess dryness, limb pain, fatigue and mental fatigue, where each of the domains normally reported as 0 (not at all) to 10 (extremely severe). The final ESSPRI score is the average of three: dryness, pain and fatigue. A reduction from baseline indicates the improvement of symptoms.
Measured: Baseline and Week 12
Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (VAS)
The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Measured: Baseline and Week 12
Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS)
The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Measured: Baseline and Week 12
Change From Baseline in Short Form (36) Health Survey (SF-36) Physical Component Score
The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Measured: Baseline and Week 12
Change From Baseline in Short Form (36) Health Survey (SF-36) Mental Component Score
The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Measured: Baseline and Week 12
Change From Baseline in Multidimensional Fatigue Inventory (MFI)
The MFI is a patient self-reported outcome measure (questionnaires) to assess fatigue covering the following dimensions: General Fatigue, Physical Fatigue, Mental Fatigue, Reduced Motivation and Reduced Activity. Each dimension has a possible range from 4-20. The reported total score has a range from 20-100. A reduction from baseline in MFI indicates improvement.
Measured: Baseline and Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Oct 201429 Jun 2018

First posted14 Nov 2014 (estimated)
Study start22 Oct 2014
Primary completion29 Jun 2018
Study completion29 Jun 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Primary Sjögren's SyndromeStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 69 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02291029
Sponsor's protocol ID: CCFZ533X2203
Status last verified: Jul 2019
First submitted: 4 Nov 2014
Last updated: 5 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Fisher BA, Szanto A, Ng WF, Bombardieri M, Posch MG, Papas AS, Farag AM, Daikeler T, Bannert B, Kyburz D, Kivitz AJ, Carsons SE, Isenberg DA, Barone F, Bowman SJ, Espie P, Floch D, Dupuy C, Ren X, Faerber PM, Wright AM, Hockey HU, Rotte M, Milojevic J, Avrameas A, Valentin MA, Rush JS, Gergely P. Assessment of the anti-CD40 antibody iscalimab in patients with primary Sjogren's syndrome: a multicentre, randomised, double-blind, placebo-controlled, proof-of-concept study. Lancet Rheumatol. 2020 Mar;2(3):e142-e152. doi: 10.1016/S2665-9913(19)30135-3. Epub 2020 Jan 23.
PubMed 38263652

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CFZ533 in Patients With Primary Sjögren's Syndrome
ClinicalTrials.gov ID: NCT02291029
Acronym / sponsor ID: CCFZ533X2203
Conditions: Primary Sjögren's Syndrome
Keywords: Sjögren's syndrome, CFZ533, ESSDAI
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 69 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 22 Oct 2014 (actual)
Primary completion: 29 Jun 2018 (actual)
Study completion: 29 Jun 2018 (actual)
First posted: 14 Nov 2014 (estimated)
Results: Posted 14 Aug 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 6

Arm	Type	Receives
CFZ533 active- Cohort 2	Experimental	Drug: CFZ533 active - Cohort 2multiple doses of CFZ533 intravenous infusion
CFZ533 placebo- Cohort 2	Placebo comparator	Drug: CFZ533 placebo - Cohort 2multiple doses of placebo intravenous infusion
CFZ533 active - Cohort 1	Experimental	Drug: CFZ533 active - Cohort 1multiple doses of CFZ533 s.c. injection
CFZ533 placebo - Cohort 1	Placebo comparator	Drug: CFZ533 placebo- Cohort 1multiple doses of placebo s.c. injection
CFZ533 Treatment Arm 1 - Cohort 3	Experimental	Drug: CFZ533 active -Cohort 3multiple doses of CFZ533 s.c. injection
CFZ533 Treatment Arm 2 - Cohort 3	Experimental	Drug: CFZ533 active - Cohort 3Single dose of CFZ533 i.v. infusion and multiple doses of CFZ533 s.c. injection

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

Diagnosis of primary Sjögren's syndrome
ESSDAI score ≥ 6

Key exclusion criteria (5 listed)

Secondary Sjögren's syndrome
Receiving cyclosphosphamide, corticosteroid bolus with dose over 1 mg/kg, rituximab, belimunab, other immunosuppressives.
At significant risk for thromboembolic event
Clinically significant systemic infection
Significant elevated risk for infection

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
CFZ533 active - Cohort 1multiple doses of CFZ533 s.c. injection	Drug	CFZ533 active - Cohort 1
CFZ533 placebo- Cohort 1multiple doses of placebo s.c. injection	Drug	CFZ533 placebo - Cohort 1
CFZ533 active - Cohort 2multiple doses of CFZ533 intravenous infusion	Drug	CFZ533 active- Cohort 2
CFZ533 placebo - Cohort 2multiple doses of placebo intravenous infusion	Drug	CFZ533 placebo- Cohort 2
CFZ533 active -Cohort 3multiple doses of CFZ533 s.c. injection	Drug	CFZ533 Treatment Arm 1 - Cohort 3
CFZ533 active - Cohort 3Single dose of CFZ533 i.v. infusion and multiple doses of CFZ533 s.c. injection	Drug	CFZ533 Treatment Arm 2 - Cohort 3

Primary outcome

Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)
Time frame: Baseline and Week 12
The effect of CFZ533 on clinical disease activity was measured by the change in ESSDAI (EULAR Sjögren's syndrome disease activity index) between baseline and week 12. The instrument 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 (range 0-123). A reduction from baseline indicates improvement in patients.

6 secondary outcomes

Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI)
Time frame: Baseline and Week 12
Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (VAS)
Time frame: Baseline and Week 12
Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS)
Time frame: Baseline and Week 12
Change From Baseline in Short Form (36) Health Survey (SF-36) Physical Component Score
Time frame: Baseline and Week 12
Change From Baseline in Short Form (36) Health Survey (SF-36) Mental Component Score
Time frame: Baseline and Week 12
Change From Baseline in Multidimensional Fatigue Inventory (MFI)
Time frame: Baseline and Week 12

Results and safety

Results posted to the registry on 14 Aug 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1 CFZ533	8	8
Cohort 1 Placebo	4	3
Cohort 2 CFZ533	21	20
Cohort 2 Placebo	11	11
Cohort 3 CFZ533 Arm 1	13	13
Cohort 3 CFZ533 Arm 2	12	12

Primary outcome: Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)

Time frame: Baseline and Week 12. Mean (Standard Deviation). Unit: units on a scale

Measure	Cohort 1 CFZ533 n = 8	Cohort 1 Placebo n = 4	Cohort 2 CFZ533 n = 21	Cohort 2 Placebo n = 11	Cohort 3 CFZ533 Arm 1 n = 13	Cohort 3 CFZ533 Arm 2 n = 12
Baseline	12.0 (3.78)	11.8 (3.86)	10.6 (4.44)	11.0 (5.16)	12.7 (6.06)	10.4 (5.87)
Week 12	9.6 (5.45)	9.8 (3.30)	4.2 (4.25)	9.7 (9.05)	7.2 (6.69)	2.8 (2.48)
Change from Baseline to Week 12	-2.4 (2.77)	-2.0 (2.45)	-6.4 (4.00)	-1.3 (8.06)	-5.5 (5.49)	-7.6 (7.14)

Cohort 1 CFZ533 vs Cohort 1 Placebo: Mean Difference (Net): -0.41 (95% CI -3.70 to 2.89), p = 0.397, Repeated measures model
Cohort 2 CFZ533 vs Cohort 2 Placebo: Mean Difference (Net): -5.21 (95% CI -9.46 to -0.96), p = 0.009, Repeated measures model
Cohort 3 CFZ533 Arm 1 vs Cohort 3 CFZ533 Arm 2: Mean Difference (Net): 2.34 (95% CI -2.78 to 7.45), p = 0.344, Repeated measures model

Population analysed: PD Population (all subjects with available Pharmacodynamics data and no protocol deviations with relevant impact on PD data)

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Cohort 1 and 2: 255 days (30 days post study completion) Cohort 3: 171 days (30 days post study completion). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1 CFZ533	1 of 8 (12.5%)	8 of 8 (100%)	0 of 8
Cohort 1 Placebo	0 of 4 (0%)	4 of 4 (100%)	0 of 4
Cohort 2 CFZ533	1 of 21 (4.8%)	11 of 21 (52.4%)	0 of 21
Cohort 2 Placebo	0 of 11 (0%)	7 of 11 (63.6%)	0 of 11
Cohort 3 CFZ533 Arm 1	0 of 13 (0%)	12 of 13 (92.3%)	0 of 13
Cohort 3 CFZ533 Arm 2	1 of 12 (8.3%)	12 of 12 (100%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 38263652Fisher BA, Szanto A, Ng WF, Bombardieri M, Posch MG, Papas AS, Farag AM, Daikeler T, Bannert B, Kyburz D, Kivitz AJ, Carsons SE, Isenberg DA, Barone F, Bowman SJ, Espie P, Floch D, Dupuy C, Ren X, Faerber PM, Wright AM, Hockey HU, Rotte M, Milojevic J, Avrameas A, Valentin MA, Rush JS, Gergely P. Assessment of the anti-CD40 antibody iscalimab in patients with primary Sjogren's syndrome: a multicentre, randomised, double-blind, placebo-controlled, proof-of-concept study. Lancet Rheumatol. 2020 Mar;2(3):e142-e152. doi: 10.1016/S2665-9913(19)30135-3. Epub 2020 Jan 23.
A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

9 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States, United Kingdom, Germany, Hungary, Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Aug 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) (time frame: Baseline and Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02291029, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.