Primary Sjögren's SyndromePhase 2NCT02775916

Safety, Pharmacokinetics, and Preliminary Efficacy Study of CDZ173 in Patients With Primary Sjögren's Syndrome

A Phase 2 study testing CDZ173 in people with Primary Sjögren's Syndrome, run by Novartis Pharmaceuticals at 2 sites in 2 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 is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173 in patients with primary Sjögren's syndrome. 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?: CDZ173
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 30 participants
Where: 2 sitesin 2 countries
Study dates: 1 Jun 2016to 17 May 2017
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 (pSS)
ESSDAI score ≥ 6 at screening visit

Who cannot join

Secondary Sjögren's syndrome
Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

Germany1 site

Novartis Investigative Site
Berlin, 10117

Other trials in Germany

Hungary1 site

Novartis Investigative Site
Debrecen, 4032

Other trials in Hungary

About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CDZ173 in Patients With Primary Sjögren's Syndrome

This Study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173 in patients with primary Sjögren's syndrome.

Read the detailed description

This study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173, a selective PI3K delta inhibitor, for 12 weeks, in patients with primary Sjögren's syndrome. Data from this study will provide the basis for further development of the compound for the treatment of primary Sjögren's syndrome.

Conditions: Primary Sjögren's Syndrome
Being tested: CDZ173, Placebo

What participants receive

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

CDZ173Drug
PlaceboDrug

Study groups in NCT02775916
Study group	Role	What they receive
CDZ173	Experimental	Capsule Receives: Drug: CDZ173
Placebo	Placebo comparator	Capsule matching Placebo Receives: Drug: Placebo

What is being measured

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

Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85
Safety and tolerability of CDZ173 in patients with primary Sjögren's syndrome up to End of Treatment Day 85
Measured: up to Day 85
Change From Baseline in the EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI) After 12 Weeks of Treatment Day 85
The ESSPRI is an established disease outcome measure for Sjögren's syndrome. The ESSPRI is a patient-reported, subjective symptom index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of three questions covering the cardinal symptoms of Sjögren's syndrome: dryness, fatigue and pain (articular and/or muscular). Each domain scored on scale of 0-10 (0 =no symptom at all and 10 = worst symptom imaginable), and an overall score is calculated as the mean of the three individual domains where all domains carry the same weight. Minimum score can be 0 and maximum score can be 10.
Measured: Baseline and 12 weeks (Day 85)

Secondary outcomes (5)

Change From Baseline in the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) After 12 Weeks of Treatment Day 85
The ESSDAI is an established disease outcome measure for Sjögren's syndrome. 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. A reduction from baseline (i.e., a negative change from baseline) in the ESSDAI score is indicative of improvement in a patient.). Each domain is assessed for activity level (i.e., no, low, moderate, high) and assigned a numerical score based on pre-determined weighting of each individual domain. An overall score is then calculated as the sum of all individual weighted domain scores. Overall score is calculated as sum of all individual weighted domain scores (ranges from 0 (best) to 123 (worst activity).
Measured: Baseline and 12 weeks (Day 85)
Change From Baseline in the Short Form (36) Health Survey (SF-36) After 12 Weeks of Treatment Day 85
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 12 weeks (Day 85)
Change in Baseline in Multidimensional Fatigue Inventory (MFI) After 12 Weeks of Treatment (Day 85)
The Multidimensional Fatigue Inventory (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.
The reported total score has a range from 20-100, higher scores are associated with greater fatigue.
Measured: Baseline and 12 weeks (Day 85)
Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (Physician VAS) After 12 Weeks of Treatment Day 85
A reduction from baseline (i.e., a negative change from baseline) in physician global VAS assessment score indicates improvement in patients. 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 12 weeks (Day 85)
Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS) After 12 Weeks of Treatment Day 85
A reduction from baseline (or, a negative change from baseline) in patient global VAS assessment score indicates improvement in patients.
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 12 weeks

Timeline

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

1 Jun 201617 May 2017

First posted18 May 2016 (estimated)
Study start1 Jun 2016
Primary completion17 May 2017
Study completion17 May 2017

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.
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: Double blind
Who is masked: Participant, Investigator
Enrolment: 30 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02775916
Sponsor's protocol ID: CCDZ173X2203
Other IDs: 2014-004616-12, 2014
Status last verified: Sep 2019
First submitted: 16 May 2016
Last updated: 5 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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?

See locations

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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 Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CDZ173 in Patients With Primary Sjögren's Syndrome
ClinicalTrials.gov ID: NCT02775916
Acronym / sponsor ID: CCDZ173X2203
Conditions: Primary Sjögren's Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 30 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 1 Jun 2016 (actual)
Primary completion: 17 May 2017 (actual)
Study completion: 17 May 2017 (actual)
First posted: 18 May 2016 (estimated)
Results: Posted 18 Oct 2019

Clinical summary

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

This Study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173 in patients with primary Sjögren's syndrome.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
CDZ173	Experimental	Drug: CDZ173Capsule
Placebo	Placebo comparator	Drug: PlaceboCapsule matching Placebo

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 (pSS)
ESSDAI score ≥ 6 at screening visit

Key exclusion criteria (2 listed)

Secondary Sjögren's syndrome
Other protocol-defined inclusion/exclusion criteria may apply.

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
CDZ173	Drug	CDZ173
Placebo	Drug	Placebo

Primary outcome

Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85
Time frame: up to Day 85
Safety and tolerability of CDZ173 in patients with primary Sjögren's syndrome up to End of Treatment Day 85
Change From Baseline in the EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI) After 12 Weeks of Treatment Day 85
Time frame: Baseline and 12 weeks (Day 85)
The ESSPRI is an established disease outcome measure for Sjögren's syndrome. The ESSPRI is a patient-reported, subjective symptom index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of three questions covering the cardinal symptoms of Sjögren's syndrome: dryness, fatigue and pain (articular and/or muscular). Each domain scored on scale of 0-10 (0 =no symptom at all and 10 = worst symptom imaginable), and an overall score is calculated as the mean of the three individual domains where all domains carry the same weight. Minimum score can be 0 and maximum score can be 10.

5 secondary outcomes

Change From Baseline in the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) After 12 Weeks of Treatment Day 85
Time frame: Baseline and 12 weeks (Day 85)
Change From Baseline in the Short Form (36) Health Survey (SF-36) After 12 Weeks of Treatment Day 85
Time frame: Baseline and 12 weeks (Day 85)
Change in Baseline in Multidimensional Fatigue Inventory (MFI) After 12 Weeks of Treatment (Day 85)
Time frame: Baseline and 12 weeks (Day 85)
Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (Physician VAS) After 12 Weeks of Treatment Day 85
Time frame: Baseline and 12 weeks (Day 85)
Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS) After 12 Weeks of Treatment Day 85
Time frame: Baseline and 12 weeks

Results and safety

Results posted to the registry on 18 Oct 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
CDZ173	20	17
Placebo	10	10

Primary outcome: Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85

Time frame: up to Day 85. Number. Unit: count of participants

Measure	CDZ173 n = 20	Placebo n = 10
Participants with at least one AE	20	8
Participants with at least one SAE	1	0
Death	0	0

Population analysed: Safety Analysis Set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the safety analysis set

Primary outcome: Change From Baseline in the EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI) After 12 Weeks of Treatment Day 85

Time frame: Baseline and 12 weeks (Day 85). Mean (Standard Deviation). Unit: total score on scale

Measure	CDZ173 n = 20	Placebo n = 10
Value	-1.778 (2.4509)	-0.741 (1.3517)

CDZ173 vs Placebo: Mean Difference (Net): -0.69 (95% CI -3.343 to 1.937)

Population analysed: PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit (Day 113). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
CDZ173	1 of 20 (5%)	19 of 20 (95%)	0 of 20
Placebo	0 of 10 (0%)	8 of 10 (80%)	0 of 10

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.
A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.
Individual participant dataSharing planned: No. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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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.

The record lists no contact. Use the official record to reach the sponsor.

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CDZ173 in people with Primary Sjögren's Syndrome, run by Novartis Pharmaceuticals at 2 sites in 2 countries.

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 2 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 2 sites in Germany, Hungary. 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 18 Oct 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?

Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85 (time frame: up to Day 85). The study lists 2 primary outcomes in all. 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 NCT02775916.

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.