Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2014-004616-12 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CDZ173 | Experimental | Capsule |
|
| Placebo | Placebo Comparator | Capsule matching Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CDZ173 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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. | Baseline and 12 weeks (Day 85) |
| Measure | Description | Time Frame |
|---|---|---|
| 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). |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Berlin | 10117 | Germany | |||
| Novartis Investigative Site |
Not provided
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on novartisclinicatrials.com | View source |
Not provided
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
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | CDZ173 | Capsule BID |
| FG001 | Placebo | Capsule matching Placebo BID |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | CDZ173 | Capsule BID |
| BG001 | Placebo | Capsule matching Placebo BID |
| BG002 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | 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 | Safety Analysis Set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the safety analysis set | Posted | Number | count of participants | up to Day 85 |
|
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)
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CDZ173 | Capsule BID | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dry Eye | Eye disorders | MedDRA (20.0) | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 8627788300 | Novartis.email@novartis.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 30, 2017 | May 14, 2018 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Sep 15, 2016 | May 14, 2018 | Prot_001.pdf |
Not provided
| ID | Term |
|---|---|
| C000625376 | leniolisib |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| 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. | 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. | 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). | 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). | Baseline and 12 weeks |
| Debrecen |
| 4032 |
| Hungary |
| Total |
Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | 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. | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Mean | Standard Deviation | total score on scale | Baseline and 12 weeks (Day 85) |
|
|
|
|
| Secondary | 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). | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Least Squares Mean | Standard Error | total score on scale | Baseline and 12 weeks (Day 85) |
|
|
|
|
| Secondary | 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. | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Least Squares Mean | Standard Error | total score on a scale | Baseline and 12 weeks (Day 85) |
|
|
|
|
| Secondary | 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. | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Least Squares Mean | Standard Error | total score on scale | Baseline and 12 weeks (Day 85) |
|
|
|
|
| Secondary | 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). | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Least Squares Mean | Standard Error | total score on a scale | Baseline and 12 weeks (Day 85) |
|
|
|
|
| Secondary | 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). | PD analysis set - All randomized patients in the CDZ173 group (20 patients) and placebo group (10 patients) were included in the PD analysis set | Posted | Least Squares Mean | Standard Error | total score on a scale | Baseline and 12 weeks |
|
|
|
|
| 20 |
| 1 |
| 20 |
| 19 |
| 20 |
| EG001 | Placebo | Capsule matching Placebo BID | 0 | 10 | 0 | 10 | 8 | 10 |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Chest Discomfort | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Chills | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Feeling Cold | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Infected Bite | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Tooth Infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Vaginal Infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
|
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Sjogren's Syndrome | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
|
| Depression | Psychiatric disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (20.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Phlebitis | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
|
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 or disclosure of trial results in their entirety.
| Mental Component Summary Score |
|
|
| Mean Difference (Net) |
| 4.33 |
| Standard Error of the Mean |
| 4.615 |
| 2-Sided |
| 95 |
| -5.27 |
| 13.93 |
| Superiority |