A Dose Finding Study to Assess the Effect of LIK066 Compared to Placebo or Empagliflozin in Patients With Type 2 Diabetes Mellitus and Heart Failure
Sponsor
Novartis Pharmaceuticals (Industry)
Overall Status
Terminated
CT.gov ID
NCT03152552
Collaborator
(none)
125
106
5
10.4
1.2
0.1
Study Details
Study Description
Brief Summary
This was a dose-finding study to evaluate the efficacy, safety and tolerability of 3 different doses of LIK066 compared to placebo or empagliflozin in T2DM patients with heart failure
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
The study was prematurely discontinued on 04-May-2018 due to slow enrollment that would preclude obtaining study results in a timely manner.
Study Design
Study Type:
Interventional
Actual Enrollment
:
125 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Primary Purpose:
Treatment
Official Title:
A Multi-center, Randomized, Double-blind, Parallel-group Dose-finding Study to Assess the Effect of 3 Doses of LIK066 Compared to Placebo or Empagliflozin in Type 2 Diabetes Mellitus Patients With Heart Failure
Actual Study Start Date
:
Jul 25, 2017
Actual Primary Completion Date
:
Jun 6, 2018
Actual Study Completion Date
:
Jun 6, 2018
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: LIK066 2.5mg Eligible participants randomized to this treatment arm received the LIK066 2.5mg dose regimen once daily for 36 weeks. |
Drug: LIK066
LIK066 was supplied in different doses as tablets taken orally.
|
| Experimental: LIK066 10mg Eligible participants randomized to this treatment arm received the LIK066 10mg dose regimen once daily for 36 weeks. |
Drug: LIK066
LIK066 was supplied in different doses as tablets taken orally.
|
| Experimental: LIK066 50mg Eligible participants randomized to this treatment arm received the LIK066 50mg dose regimen once daily for 36 weeks. |
Drug: LIK066
LIK066 was supplied in different doses as tablets taken orally.
|
| Active Comparator: Empagliflozin Participants randomized to this treatment arm received empagliflozin once daily for 36 weeks. |
Drug: Empagliflozin
Empagliflozin was supplied as capsules taken orally.
|
| Placebo Comparator: Placebo Participants randomized to this treatment arm received LIK066 matching placebo and empagliflozin matching placebo. |
Drug: Placebo
Placebo was supplied as tablets and capsules taken orally.
|
Outcome Measures
Primary Outcome Measures
- Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 12 [Baseline, Week 12]
Evaluation of NT-proBNP was performed by a central laboratory. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this primary endpoint due to early study termination. Only descriptive statistics are presented.
Secondary Outcome Measures
- Change From Baseline in Glycated Hemoglobin (HbA1c) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
HbA1c was measured from a blood sample and analyzed using a National Glycohemoglobin Standardization Program (NGSP) certified method at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Fasting Plasma Glucose (FPG) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
FPG was measured from a blood sample after an overnight fast; patients were not allowed to eat or drink anything (except water) for at least 8 h before each study visit. Samples were analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Body Weight at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Body weight was measured to the nearest 0.1 kg on a calibrated scale (weight and bio-impedance measurements), provided by the sponsor. Exceptionally (e.g. if the body weight exceeded the limits of the provided scale) sites were allowed to use another scale for weight measurement as available, but during the study the same scale was to be used for the same patient. The measurement was performed with the study patient in underwear and without shoes. Indoor clothing was also acceptable, but measurements were to be done consistently (either with underwear or with indoor clothing) throughout the study. Voiding before weight measurement was required. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Body Composition Assessed by Bio-impedance (Total Body Fat Mass) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented
- Change From Baseline in Body Composition Assessed by Bio-impedance (Visceral Fat Level) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. Visceral fat levels were measured by Omron device. Levels ranged from 1 - 30 and are relative (not absolute) values. The Omron scale values are: 0 - 9 (normal), 10 - 14 (high) and 15 - 30 (very high). Visceral fat area ( 0 - approx. 300cm^2, 1 inch = 2.54 cm) distribution with 30 levels.
- Change From Baseline in Body Composition Assessed by Bio-impedance (Lean Body Mass) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented
- Change From Baseline in Body Composition Assessed by DXA (Total Body Fat Mass) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A whole body DXA scan was performed to assess Total Body Fat Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done.
- Change From Baseline in Body Composition Assessed by DXA (Visceral Fat Mass) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A whole body DXA scan was performed to assess Visceral Fat Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done.
- Change From Baseline in Body Composition Assessed by DXA (Lean Body Mass) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A whole body DXA scan was performed to assess Lean Body Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done.
- Change From Baseline in Body Composition Assessed by DXA (Total Body Water) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A whole body DXA scan was performed to assess Total Body Water (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done.
- Change From Baseline in Sitting Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Three sitting BP measurements were performed. At each visit, sitting BP was derived as the mean of three readings of the sitting SBP/DBP at that visit. Pre-planned statistical analyses were not performed for these secondary endpoints due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Fasting Lipid Profile (Triglycerides (TG)) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
TG was measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Fasting Lipid Profile (Lipoproteins) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Lipoproteins (High Density Lipoprotein (HDL) Cholesterol, Low Density Lipoprotein (LDL) Cholesterol) were measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis were not performed for these secondary endpoints due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Fasting Lipid Profile (Total Cholesterol) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Total Cholesterol was measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in High Sensitive C-reactive Protein (hsCRP) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
hs-CRP is an inflammation biomarker. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in 24 Hour Urinary Glucose Excretion (UGE) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
UGE was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive analysis done.
- Change From Baseline in 24 Hour Sodium Excretion at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Sodium excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive statistics were done.
- Change From Baseline in Left Atrial Size at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A limited two-dimensional and Doppler ECHO examination was performed to assess ECHO parameters. The images were sent to a central reading vendor for independent review and analysis. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in Left Atrial Volume at Weeks 12 and 36 [Baseline, Week 12, Week 36]
A limited two-dimensional and Doppler ECHO examination was performed to assess ECHO parameters. The images were sent to a central reading vendor for independent review and analysis.Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Number of Participants With New York Heart Association (NYHA) Class I, II, II or IV [Baseline, Week 12, Week 36]
The NYHA Functional Classification classifies patients' heart failure according to the severity of their symptoms. The classification is as follows: Class I: no limitation of physical activity, ordinary physical activity does not cause undue fatigue, palpitation, or dyspnea (shortness of breath); Class II: slight limitation to physical activity, comfortable at rest, ordinary physical activity results in fatigue, palpitation or dyspnea; Class III: marked limitation of physical activity, comfortable at rest, less than ordinary activity causes fatigue, palpitation or dyspnea; Class IV: unable to carry on any physical activity without discomfort, symptoms of heart failure at rest, if any physical activity is undertaken, discomfort increases. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Week 12 and 36 [Week 12, Week 36]
The change from BL in NYHA class at a given visit is a three-category ordinal variable (improved/unchanged/worsened) with the following definition: 1. Improved, if NYHA class decreases at least one level from BL; 2. Unchanged, if NYHA class is unchanged from BL; 3. Worsened, if NYHA class increases at least one level from BL. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 36 [Baseline, Week 36]
Evaluation of NT-proBNP was performed by a central laboratory. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented.
- Change From Baseline in 24 Hour Urinary Calcium Excretion at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Urinary calcium excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive analysis done.
- 24 Hour Urinary Phosphate Excretion at Weeks 12 and 36 [Baseline, Week 12, Week 36]
Urinary phosphate excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive statistics were done.
- Change From Baseline in Bone Mineral Density (BMD) at Weeks 12 and 36 [Baseline, Week 12, Week 36]
To evaluate bone mineral density as assessed by bone mineral content after 12 weeks and after 36 weeks of treatment. Only descriptive statistics were done.
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
-
BMI ≥ 22kg/m^2
-
Type 2 diabetes with HbA1c between 6.5% and 10.0%
-
Documented symptomatic chronic heart failure (NYHA II-IV)
-
Plasma NT-proBNP > 300pg/ml
-
eGFR ≥ 45ml/min/1.73m^2 (calculated by MDRD)
Key Exclusion Criteria:
-
Pregnant or nursing (lactating) women
-
Type 1 diabetes, monogenic diabetes, diabetes resulting from pancreatic injury, or secondary forms of diabetes
-
History of ketoacidosis, lactic acidosis, or hyperosmolar coma
-
Symptomatic genital infection or UTI within 4 weeks of screening
-
Myocardial infarction, stroke, surgery for heart disease, percutaneous coronary intervention within 3 months of randomization
-
Unstable angina within 3 months of screening
-
Isolated right HF due to pulmonary disease
-
Patients with a mean sitting systolic blood pressure ≤ 100mmHg, at randomization
-
History of lower limb amputation
-
Diabetic foot ulcer at screening
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Novartis Investigative Site | Huntsville | Alabama | United States | 35801 |
| 2 | Novartis Investigative Site | Carmichael | California | United States | 95608 |
| 3 | Novartis Investigative Site | Concord | California | United States | 94520 |
| 4 | Novartis Investigative Site | Long Beach | California | United States | 90813 |
| 5 | Novartis Investigative Site | Northridge | California | United States | 91325 |
| 6 | Novartis Investigative Site | Stockton | California | United States | 95204 |
| 7 | Novartis Investigative Site | Colorado Springs | Colorado | United States | 80906 |
| 8 | Novartis Investigative Site | Bradenton | Florida | United States | 34209 |
| 9 | Novartis Investigative Site | Clearwater | Florida | United States | 33756 |
| 10 | Novartis Investigative Site | Delray Beach | Florida | United States | 33446 |
| 11 | Novartis Investigative Site | Fort Lauderdale | Florida | United States | 33312 |
| 12 | Novartis Investigative Site | Gurnee | Illinois | United States | 60031 |
| 13 | Novartis Investigative Site | Bogalusa | Louisiana | United States | 70427 |
| 14 | Novartis Investigative Site | Jackson | Mississippi | United States | 39209 |
| 15 | Novartis Investigative Site | Saint Louis | Missouri | United States | 63128 |
| 16 | Novartis Investigative Site | Omaha | Nebraska | United States | 68114 |
| 17 | Novartis Investigative Site | Charleston | South Carolina | United States | 29407 |
| 18 | Novartis Investigative Site | Columbia | South Carolina | United States | 29203 |
| 19 | Novartis Investigative Site | San Antonio | Texas | United States | 78229 |
| 20 | Novartis Investigative Site | Sugar Land | Texas | United States | 77479 |
| 21 | Novartis Investigative Site | Tacoma | Washington | United States | 98405 |
| 22 | Novartis Investigative Site | Caba | Buenos Aires | Argentina | 1407 |
| 23 | Novartis Investigative Site | Caba | Buenos Aires | Argentina | C1056ABJ |
| 24 | Novartis Investigative Site | Caba | Capital Federal | Argentina | C1179AAB |
| 25 | Novartis Investigative Site | Buenos Aires | Argentina | C1120AAC | |
| 26 | Novartis Investigative Site | Graz | Austria | A-8036 | |
| 27 | Novartis Investigative Site | Wien | Austria | 1090 | |
| 28 | Novartis Investigative Site | Wien | Austria | 1130 | |
| 29 | Novartis Investigative Site | Lennik | Brussels | Belgium | 1070 |
| 30 | Novartis Investigative Site | Aalst | Belgium | 9300 | |
| 31 | Novartis Investigative Site | Bonheiden | Belgium | 2820 | |
| 32 | Novartis Investigative Site | Leuven | Belgium | 3000 | |
| 33 | Novartis Investigative Site | Liege | Belgium | 4000 | |
| 34 | Novartis Investigative Site | Sofia | Bulgaria | 1233 | |
| 35 | Novartis Investigative Site | Sofia | Bulgaria | 1309 | |
| 36 | Novartis Investigative Site | Sofia | Bulgaria | 1431 | |
| 37 | Novartis Investigative Site | Sofia | Bulgaria | 1709 | |
| 38 | Novartis Investigative Site | London | Ontario | Canada | N6A 5A5 |
| 39 | Novartis Investigative Site | Toronto | Ontario | Canada | M5B 1W8 |
| 40 | Novartis Investigative Site | Montreal | Quebec | Canada | H4A 3J1 |
| 41 | Novartis Investigative Site | Sainte Foy | Quebec | Canada | G1V 4G5 |
| 42 | Novartis Investigative Site | St-Jerome | Quebec | Canada | J7Z 5T3 |
| 43 | Novartis Investigative Site | Quebec | Canada | G1V 4G2 | |
| 44 | Novartis Investigative Site | Krapinske toplice | Croatia | 49 217 | |
| 45 | Novartis Investigative Site | Rijeka | Croatia | 51000 | |
| 46 | Novartis Investigative Site | Zagreb | Croatia | 10000 | |
| 47 | Novartis Investigative Site | Brandys nad Labem | Czech Republic | Czechia | 250 01 |
| 48 | Novartis Investigative Site | Svitavy | Czech Republic | Czechia | 568 25 |
| 49 | Novartis Investigative Site | Trebic | Czech Republic | Czechia | 674 01 |
| 50 | Novartis Investigative Site | Karvina | Czechia | 73506 | |
| 51 | Novartis Investigative Site | Kolin | Czechia | 280 20 | |
| 52 | Novartis Investigative Site | Prague 5 | Czechia | 158 00 | |
| 53 | Novartis Investigative Site | Praha | Czechia | 12808 | |
| 54 | Novartis Investigative Site | Prerov | Czechia | 751 52 | |
| 55 | Novartis Investigative Site | Hellerup | Denmark | 2900 | |
| 56 | Novartis Investigative Site | Svendborg | Denmark | 5700 | |
| 57 | Novartis Investigative Site | Bad Oeynhausen | Germany | 32545 | |
| 58 | Novartis Investigative Site | Berlin | Germany | 10789 | |
| 59 | Novartis Investigative Site | Berlin | Germany | 12157 | |
| 60 | Novartis Investigative Site | Berlin | Germany | 13347 | |
| 61 | Novartis Investigative Site | Frankfurt | Germany | 60594 | |
| 62 | Novartis Investigative Site | Halle | Germany | 06120 | |
| 63 | Novartis Investigative Site | Hamburg | Germany | 20099 | |
| 64 | Novartis Investigative Site | Stuttgart | Germany | 70378 | |
| 65 | Novartis Investigative Site | Budapest | HUN | Hungary | 1145 |
| 66 | Novartis Investigative Site | Budapest | Hungary | 1134 | |
| 67 | Novartis Investigative Site | Szeged | Hungary | 6720 | |
| 68 | Novartis Investigative Site | Szekszard | Hungary | 7100 | |
| 69 | Novartis Investigative Site | Wilton | Cork | Ireland | |
| 70 | Novartis Investigative Site | County Limerick | Ireland | V94 F858 | |
| 71 | Novartis Investigative Site | Dublin 4 | Ireland | ||
| 72 | Novartis Investigative Site | Bergamo | BG | Italy | 24127 |
| 73 | Novartis Investigative Site | Milano | MI | Italy | 20132 |
| 74 | Novartis Investigative Site | San Donato Milanese | MI | Italy | 20097 |
| 75 | Novartis Investigative Site | Milano | Italy | 20149 | |
| 76 | Novartis Investigative Site | Rimini | Italy | 47923 | |
| 77 | Novartis Investigative Site | Cheongju si | Chungcheongbuk Do | Korea, Republic of | 28644 |
| 78 | Novartis Investigative Site | Wonju | Gangwon-Do | Korea, Republic of | 26426 |
| 79 | Novartis Investigative Site | Bundang Gu | Gyeonggi Do | Korea, Republic of | 13620 |
| 80 | Novartis Investigative Site | Seoul | Korea, Republic of | 03080 | |
| 81 | Novartis Investigative Site | Durango | Mexico | 34000 | |
| 82 | Novartis Investigative Site | Alkmaar | Netherlands | 1815 JD | |
| 83 | Novartis Investigative Site | Groningen | Netherlands | 9713 GZ | |
| 84 | Novartis Investigative Site | Utrecht | Netherlands | 3584 CX | |
| 85 | Novartis Investigative Site | Venlo | Netherlands | 5912 BL | |
| 86 | Novartis Investigative Site | Loerenskog | Norway | NO 1478 | |
| 87 | Novartis Investigative Site | Oslo | Norway | 0372 | |
| 88 | Novartis Investigative Site | Trondheim | Norway | 7006 | |
| 89 | Novartis Investigative Site | Warszawa | Poland | 00-874 | |
| 90 | Novartis Investigative Site | Wroclaw | Poland | 51-314 | |
| 91 | Novartis Investigative Site | Ponce | Puerto Rico | 00717 | |
| 92 | Novartis Investigative Site | Singapore | Singapore | 169609 | |
| 93 | Novartis Investigative Site | Bloemfontein | Free State | South Africa | 9301 |
| 94 | Novartis Investigative Site | Paarl | Western Cape | South Africa | 7626 |
| 95 | Novartis Investigative Site | Worcester | South Africa | 6850 | |
| 96 | Novartis Investigative Site | Sevilla | Andalucia | Spain | 41014 |
| 97 | Novartis Investigative Site | Villamartin | Cadiz | Spain | 11650 |
| 98 | Novartis Investigative Site | Valencia | Comunidad Valenciana | Spain | 46010 |
| 99 | Novartis Investigative Site | Caceres | Extremadura | Spain | 10003 |
| 100 | Novartis Investigative Site | Changhua | Taiwan | 50006 | |
| 101 | Novartis Investigative Site | Taichung | Taiwan | 40447 | |
| 102 | Novartis Investigative Site | Taipei | Taiwan | 11217 | |
| 103 | Novartis Investigative Site | Chelmsford | Essex | United Kingdom | CM1 7ET |
| 104 | Novartis Investigative Site | London | GBR | United Kingdom | EC1M 6BQ |
| 105 | Novartis Investigative Site | Sunderland | Tyne And Wear | United Kingdom | SR4 7TP |
| 106 | Novartis Investigative Site | Birmingham | United Kingdom | B15 2TH |
Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03152552
Other Study ID Numbers:
- CLIK066B2204
- 2016-003084-19
First Posted:
May 15, 2017
Last Update Posted:
Aug 28, 2019
Last Verified:
Aug 1, 2019
Individual Participant Data (IPD) Sharing Statement:
Undecided
Plan to Share IPD:
Undecided
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Novartis Pharmaceuticals
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | Patients were randomized in a 1:1:2:2:2 ratio to one of 5 regimens (LIK066 2.5mg, 10mg, 50 mg, EMPA 25mg, Pbo) at Visit 201 (randomization) with a plan to be treated for 36 weeks. |
|---|---|
| Pre-assignment Detail | A placebo run-in period (Epoch 2) running up to 2 weeks before randomization was used to assess eligibility |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Period Title: Treatment Period 1 (12 Weeks) | |||||
| STARTED | 15 | 16 | 31 | 30 | 33 |
| Full Analysis Set (FAS) | 15 | 16 | 30 | 30 | 33 |
| COMPLETED | 7 | 5 | 10 | 10 | 12 |
| NOT COMPLETED | 8 | 11 | 21 | 20 | 21 |
| Period Title: Treatment Period 1 (12 Weeks) | |||||
| STARTED | 7 | 5 | 9 | 9 | 11 |
| COMPLETED | 1 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 6 | 5 | 9 | 9 | 11 |
Baseline Characteristics
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo | Total |
|---|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo | Total of all reporting groups |
| Overall Participants | 15 | 16 | 30 | 30 | 33 | 124 |
| Age (Years) [Mean (Standard Deviation) ] | ||||||
| Mean (Standard Deviation) [Years] |
68.2
(7.10)
|
69.8
(9.69)
|
65.8
(9.08)
|
68.6
(7.89)
|
67.8
(10.93)
|
67.8
(9.17)
|
| Sex: Female, Male (Count of Participants) | ||||||
| Female |
1
6.7%
|
4
25%
|
6
20%
|
10
33.3%
|
14
42.4%
|
35
28.2%
|
| Male |
14
93.3%
|
12
75%
|
24
80%
|
20
66.7%
|
19
57.6%
|
89
71.8%
|
| Race/Ethnicity, Customized (Number) [Number] | ||||||
| Caucasian |
14
93.3%
|
14
87.5%
|
28
93.3%
|
28
93.3%
|
29
87.9%
|
113
91.1%
|
| Black |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
1
3%
|
1
0.8%
|
| Asian |
1
6.7%
|
2
12.5%
|
2
6.7%
|
1
3.3%
|
3
9.1%
|
9
7.3%
|
| Other |
0
0%
|
0
0%
|
0
0%
|
1
3.3%
|
0
0%
|
1
0.8%
|
Outcome Measures
| Title | Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 12 |
|---|---|
| Description | Evaluation of NT-proBNP was performed by a central laboratory. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this primary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms are considered per study primary objective. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 12 | 16 |
| Geometric Mean (95% Confidence Interval) [ratio] |
0.8
|
0.6
|
0.8
|
1.1
|
| Title | Change From Baseline in Glycated Hemoglobin (HbA1c) at Weeks 12 and 36 |
|---|---|
| Description | HbA1c was measured from a blood sample and analyzed using a National Glycohemoglobin Standardization Program (NGSP) certified method at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 12 | 14 | 18 |
| Change from BL at Week 12 |
-0.29
(0.836)
|
-0.01
(0.508)
|
-0.58
(0.335)
|
-0.44
(1.176)
|
-0.04
(0.913)
|
| Change from BL at Week 36 |
0.13
(0.961)
|
-0.60
(NA)
|
-1.83
(0.321)
|
| Title | Change From Baseline in Fasting Plasma Glucose (FPG) at Weeks 12 and 36 |
|---|---|
| Description | FPG was measured from a blood sample after an overnight fast; patients were not allowed to eat or drink anything (except water) for at least 8 h before each study visit. Samples were analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 8 | 6 | 12 | 13 | 15 |
| Change from BL at Week 12 |
-1.021
(1.0368)
|
-2.041
(4.9772)
|
-0.426
(2.1451)
|
-1.303
(2.4386)
|
-1.187
(3.9653)
|
| Change from BL at Week 36 |
0.392
(1.1119)
|
-1.200
(NA)
|
-4.733
(0.3055)
|
| Title | Change From Baseline in Body Weight at Weeks 12 and 36 |
|---|---|
| Description | Body weight was measured to the nearest 0.1 kg on a calibrated scale (weight and bio-impedance measurements), provided by the sponsor. Exceptionally (e.g. if the body weight exceeded the limits of the provided scale) sites were allowed to use another scale for weight measurement as available, but during the study the same scale was to be used for the same patient. The measurement was performed with the study patient in underwear and without shoes. Indoor clothing was also acceptable, but measurements were to be done consistently (either with underwear or with indoor clothing) throughout the study. Voiding before weight measurement was required. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 13 | 14 | 18 |
| Change from BL at Week 12 |
-0.78
(2.734)
|
-1.83
(1.402)
|
-2.15
(2.397)
|
-2.25
(1.894)
|
-0.34
(2.115)
|
| Change from BL at Week 36 |
-2.21
(1.586)
|
-3.90
(NA)
|
0.47
(6.158)
|
| Title | Change From Baseline in Body Composition Assessed by Bio-impedance (Total Body Fat Mass) at Weeks 12 and 36 |
|---|---|
| Description | Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 6 | 7 | 11 | 12 | 15 |
| Wk 12 Chge from BL |
-0.77
(2.276)
|
-1.51
(5.048)
|
-0.32
(4.675)
|
1.63
(3.639)
|
-1.77
(7.812)
|
| Wk 36 Chge from BL |
2.25
(1.485)
|
0.20
(NA)
|
6.70
(20.082)
|
| Title | Change From Baseline in Body Composition Assessed by Bio-impedance (Visceral Fat Level) at Weeks 12 and 36 |
|---|---|
| Description | Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. Visceral fat levels were measured by Omron device. Levels ranged from 1 - 30 and are relative (not absolute) values. The Omron scale values are: 0 - 9 (normal), 10 - 14 (high) and 15 - 30 (very high). Visceral fat area ( 0 - approx. 300cm^2, 1 inch = 2.54 cm) distribution with 30 levels. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 7 | 7 | 11 | 12 | 15 |
| Wk 12 Chge from BL |
-2.429
(4.6853)
|
-2.857
(3.8914)
|
-0.436
(4.6877)
|
-0.417
(1.3114)
|
-3.200
(4.7988)
|
| Wk 36 Chge from BL |
0.000
(1.4142)
|
0.000
(NA)
|
3.500
(7.7782)
|
| Title | Change From Baseline in Body Composition Assessed by Bio-impedance (Lean Body Mass) at Weeks 12 and 36 |
|---|---|
| Description | Body composition was measured in all patients using bio-impedance, except in patients where it was contra-indicated, e.g. those using an implantable cardioverter-defibrillator. Body composition parameters were assessed as available for the different models of calibrated bio-impedance scales. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 6 | 6 | 10 | 12 | 14 |
| Wk 12 Chge from BL |
-2.32
(7.063)
|
-2.32
(5.774)
|
-0.24
(2.022)
|
-0.68
(2.454)
|
1.64
(4.584)
|
| Wk 36 Chge from BL |
-0.85
(1.344)
|
-0.30
(NA)
|
-5.35
(13.223)
|
| Title | Change From Baseline in Body Composition Assessed by DXA (Total Body Fat Mass) at Weeks 12 and 36 |
|---|---|
| Description | A whole body DXA scan was performed to assess Total Body Fat Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. The analysis included patients who participated in the DXA sub-study. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 1 | 0 | 4 | 4 | 1 |
| BL Whole Body Minus Head Hologic |
35.970
(NA)
|
18.870
(NA)
|
27.550
(NA)
|
||
| Wk 12 Whole Body - Hd Hologic Chge BL |
-0.310
(NA)
|
-4.280
(NA)
|
|||
| Wk 36 Whole Body - Hd Hologic Chge BL |
-3.800
(NA)
|
-5.590
(NA)
|
|||
| BL Whole Body Minus Head Lunar |
29.350
(4.9403)
|
37.455
(6.0175)
|
|||
| Wk 12 Whole Body - Hd Lunar Chge BL |
-1.260
(NA)
|
1.190
(NA)
|
| Title | Change From Baseline in Body Composition Assessed by DXA (Visceral Fat Mass) at Weeks 12 and 36 |
|---|---|
| Description | A whole body DXA scan was performed to assess Visceral Fat Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. The analysis included patients who participated in the DXA sub-study. Due to early termination of the study, no data was collected. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 0 | 0 | 0 | 0 | 0 |
| Title | Change From Baseline in Body Composition Assessed by DXA (Lean Body Mass) at Weeks 12 and 36 |
|---|---|
| Description | A whole body DXA scan was performed to assess Lean Body Mass (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. The analysis included patients who participated in the DXA sub-study. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 1 | 0 | 4 | 4 | 1 |
| Wk 12 Whole Body - Hd Hologic Chge BL |
-1.910
(NA)
|
4.980
(NA)
|
|||
| Wk 36 Whole Body - Hd Hologic Chge BL |
0.860
(NA)
|
1.700
(NA)
|
|||
| Wk 12 Whole Body - Hd Lunar Chge BL |
-1.290
(NA)
|
-2.960
(NA)
|
| Title | Change From Baseline in Body Composition Assessed by DXA (Total Body Water) at Weeks 12 and 36 |
|---|---|
| Description | A whole body DXA scan was performed to assess Total Body Water (Whole Body Minus Head Hologic, Whole Body Minus Head Lunar). DXA data were transferred to a central reading vendor for independent review and analysis. Only descriptive statistics done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS consisted of all randomized patients who were not mis-randomized. Analysis included patients who participated in the DXA sub-study. Due to early termination of the study, no data was collected. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 0 | 0 | 0 | 0 | 0 |
| Title | Change From Baseline in Sitting Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at Weeks 12 and 36 |
|---|---|
| Description | Three sitting BP measurements were performed. At each visit, sitting BP was derived as the mean of three readings of the sitting SBP/DBP at that visit. Pre-planned statistical analyses were not performed for these secondary endpoints due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 13 | 14 | 18 |
| SBP Change from BL at Week 12 |
5.15
(13.485)
|
0.17
(15.373)
|
-9.54
(16.884)
|
-6.98
(15.031)
|
-2.85
(11.967)
|
| SBP Change from BL at Week 36 |
13.78
(17.900)
|
-4.00
(NA)
|
0.00
(8.627)
|
||
| DBP Change from BL at Week 12 |
-2.00
(6.582)
|
4.50
(12.746)
|
-4.46
(11.238)
|
-1.81
(10.421)
|
-2.00
(8.596)
|
| DBP Change from BL at Week 36 |
1.12
(3.975)
|
3.66
(NA)
|
-0.44
(8.517)
|
| Title | Change From Baseline in Fasting Lipid Profile (Triglycerides (TG)) at Weeks 12 and 36 |
|---|---|
| Description | TG was measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 6 | 12 | 13 | 15 |
| % Change from BL at Week 12 |
-1.623
(35.2838)
|
19.089
(31.4798)
|
9.878
(30.3065)
|
8.865
(35.0872)
|
-2.979
(25.1049)
|
| % Change from BL at Week 36 |
4.324
(31.4438)
|
14.286
(NA)
|
-1.111
(18.3586)
|
| Title | Change From Baseline in Fasting Lipid Profile (Lipoproteins) at Weeks 12 and 36 |
|---|---|
| Description | Lipoproteins (High Density Lipoprotein (HDL) Cholesterol, Low Density Lipoprotein (LDL) Cholesterol) were measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis were not performed for these secondary endpoints due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 7 | 12 | 12 |
| HDL % Change from BL at Week 12 |
9.33
(16.735)
|
-10.54
(20.590)
|
0.26
(9.772)
|
2.18
(12.179)
|
-0.67
(13.322)
|
| HDL % Change from BL at Week 36 |
10.70
(16.257)
|
0.00
(NA)
|
35.00
(49.497)
|
||
| LDL % Change from BL at Week 12 |
22.02
(35.466)
|
2.62
(17.525)
|
16.40
(36.928)
|
22.24
(35.145)
|
-1.59
(31.970)
|
| LDL % Change from BL at Week 36 |
22.73
(31.690)
|
-3.57
(NA)
|
0.22
(13.163)
|
| Title | Change From Baseline in Fasting Lipid Profile (Total Cholesterol) at Weeks 12 and 36 |
|---|---|
| Description | Total Cholesterol was measured on blood samples obtained after an overnight fast and analyzed at a central laboratory. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS consisted of all randomized patients who were not mis-randomized. Analysis included patients who participated in the study. Due to early termination of the study, only the data shown was available. 'Overall Number of Participants Analyzed' = enrolled in the study. 'Number Analyzed' = number of participants with data available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 7 | 12 | 12 |
| % Change from BL at Week 12 |
9.69
(23.892)
|
-2.66
(13.202)
|
6.32
(22.667)
|
10.83
(11.330)
|
1.46
(16.741)
|
| % Change from BL at Week 36 |
14.72
(13.147)
|
2.04
(NA)
|
10.27
(28.326)
|
| Title | Change From Baseline in High Sensitive C-reactive Protein (hsCRP) at Weeks 12 and 36 |
|---|---|
| Description | hs-CRP is an inflammation biomarker. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 7 | 7 | 7 | 11 | 10 |
| Change from BL at Week 12 |
0.543
|
0.722
|
1.997
|
0.714
|
1.018
|
| Change from BL at Week 36 |
0.953
|
0.620
|
0.578
|
| Title | Change From Baseline in 24 Hour Urinary Glucose Excretion (UGE) at Weeks 12 and 36 |
|---|---|
| Description | UGE was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive analysis done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. 'Overall Number of Participants Analyzed' = enrolled in the 24 hour urine collection sub-study. 'Number Analyzed' = number of participants with data available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 2 | 2 | 1 | 2 | 5 |
| Change from BL at Week 12 |
256.245
(129.0682)
|
346.360
(107.3671)
|
305.110
(NA)
|
254.270
(198.8243)
|
84.778
(222.6565)
|
| Title | Change From Baseline in 24 Hour Sodium Excretion at Weeks 12 and 36 |
|---|---|
| Description | Sodium excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive statistics were done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS consisted of all randomized patients who were not mis-randomized. Due to early termination of the study, only the data shown was available. 'Overall Number of Participants Analyzed' = enrolled in the 24 hour urine collection sub-study. 'Number Analyzed' = number of participants with data available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 2 | 2 | 2 | 2 | 7 |
| Change from BL at Week 12 |
-38.5
(86.69)
|
45.6
(40.52)
|
-42.6
(28.50)
|
82.3
(98.29)
|
-43.9
(112.48)
|
| Title | Change From Baseline in Left Atrial Size at Weeks 12 and 36 |
|---|---|
| Description | A limited two-dimensional and Doppler ECHO examination was performed to assess ECHO parameters. The images were sent to a central reading vendor for independent review and analysis. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms were considered per study objective. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 3 | 4 | 7 | 11 |
| Change from BL at Week 12 |
-1.167
(14.8123)
|
0.075
(6.7884)
|
2.700
(7.2155)
|
-1.045
(11.0223)
|
| Change from BL at Week 36 |
16.333
(20.9194)
|
0.300
(NA)
|
5.100
(6.2960)
|
| Title | Change From Baseline in Left Atrial Volume at Weeks 12 and 36 |
|---|---|
| Description | A limited two-dimensional and Doppler ECHO examination was performed to assess ECHO parameters. The images were sent to a central reading vendor for independent review and analysis.Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms were considered per study objective. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 5 | 4 | 8 | 11 |
| Change from BL at Week 12 |
12.360
(42.7067)
|
0.225
(15.4157)
|
7.725
(16.9351)
|
-3.591
(22.8382)
|
| Change from BL at Week 36 |
34.800
(51.0409)
|
-0.900
(NA)
|
11.333
(12.7892)
|
| Title | Number of Participants With New York Heart Association (NYHA) Class I, II, II or IV |
|---|---|
| Description | The NYHA Functional Classification classifies patients' heart failure according to the severity of their symptoms. The classification is as follows: Class I: no limitation of physical activity, ordinary physical activity does not cause undue fatigue, palpitation, or dyspnea (shortness of breath); Class II: slight limitation to physical activity, comfortable at rest, ordinary physical activity results in fatigue, palpitation or dyspnea; Class III: marked limitation of physical activity, comfortable at rest, less than ordinary activity causes fatigue, palpitation or dyspnea; Class IV: unable to carry on any physical activity without discomfort, symptoms of heart failure at rest, if any physical activity is undertaken, discomfort increases. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms were considered per study objective. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 13 | 18 |
| Class l |
1
6.7%
|
1
6.3%
|
1
3.3%
|
1
3.3%
|
| Class ll |
6
40%
|
6
37.5%
|
10
33.3%
|
13
43.3%
|
| Class lll |
2
13.3%
|
1
6.3%
|
2
6.7%
|
4
13.3%
|
| Class lV |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Class l |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Class ll |
3
20%
|
0
0%
|
1
3.3%
|
3
10%
|
| Class lll |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Class lV |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Title | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Week 12 and 36 |
|---|---|
| Description | The change from BL in NYHA class at a given visit is a three-category ordinal variable (improved/unchanged/worsened) with the following definition: 1. Improved, if NYHA class decreases at least one level from BL; 2. Unchanged, if NYHA class is unchanged from BL; 3. Worsened, if NYHA class increases at least one level from BL. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms were considered per study objective. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 9 | 8 | 13 | 18 |
| Improved |
1
6.7%
|
1
6.3%
|
1
3.3%
|
4
13.3%
|
| Unchanged |
8
53.3%
|
7
43.8%
|
12
40%
|
13
43.3%
|
| Worsened |
0
0%
|
0
0%
|
0
0%
|
1
3.3%
|
| Improved |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unchanged |
3
20%
|
0
0%
|
1
3.3%
|
3
10%
|
| Worsened |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Title | Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 36 |
|---|---|
| Description | Evaluation of NT-proBNP was performed by a central laboratory. For Change from baseline, Geometric mean is the geometric mean of the endpoint to baseline ratio. Pre-planned statistical analysis was not performed for this secondary endpoint due to early study termination. Only descriptive statistics are presented. |
| Time Frame | Baseline, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized patients who were not mis-randomized. LIK066 doses and placebo arms are considered per study objective. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | Placebo |
|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 3 | 0 | 1 | 3 |
| Geometric Mean (95% Confidence Interval) [ratio] |
0.7
|
1.3
|
1.0
|
| Title | Change From Baseline in 24 Hour Urinary Calcium Excretion at Weeks 12 and 36 |
|---|---|
| Description | Urinary calcium excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive analysis done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety Set: All patients who received at least 1 dose of double-blind study drug & included patients who participated in sub-study. Due to early termination of study, only data shown was available. 'Overall Number of Participants Analyzed' = enrolled in 24h urine collection sub-study. 'Number Analyzed '= number of participants with data available |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 1 | 1 | 2 | 1 | 5 |
| Change from BL at Week 12 |
1.40
(NA)
|
3.80
(NA)
|
0.10
(0.566)
|
0.60
(NA)
|
-0.49
(3.202)
|
| Title | 24 Hour Urinary Phosphate Excretion at Weeks 12 and 36 |
|---|---|
| Description | Urinary phosphate excretion was measured from a 24h urine collection from about 25% of randomized patients and analyzed at a central laboratory. Only descriptive statistics were done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Safety Set (SAF), which consisted of all patients who received at least one dose of double-blind study drug, was considered. The analysis included patients who participated in the 24h urine collection sub-study. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 2 | 2 | 2 | 1 | 6 |
| Change from BL at Week 12 |
55.35
(25.809)
|
19.25
(55.225)
|
-125.95
(105.571)
|
5.30
(NA)
|
26.07
(142.536)
|
| Title | Change From Baseline in Bone Mineral Density (BMD) at Weeks 12 and 36 |
|---|---|
| Description | To evaluate bone mineral density as assessed by bone mineral content after 12 weeks and after 36 weeks of treatment. Only descriptive statistics were done. |
| Time Frame | Baseline, Week 12, Week 36 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Safety Set (SAF), which consisted of all patients who received at least one dose of double-blind study drug, was considered. The analysis included participants who participated in the DXA sub-study. Due to early termination of the study, only the data shown was available. |
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo |
|---|---|---|---|---|---|
| Arm/Group Description | LIK066 2.5mg once daily | LIk066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo |
| Measure Participants | 1 | 0 | 1 | 1 | 1 |
| Wk 12 Whole Body - Hd Hologic Chge BL |
-13.250
(NA)
|
-3.340
(NA)
|
|||
| Wk 36 Whole Body - Hd Hologic Chge BL |
-58.220
(NA)
|
64.620
(NA)
|
|||
| Wk 12 Whole Body - Hd Lunar Chge BL |
-78.750
(NA)
|
37.350
(NA)
|
Adverse Events
| Time Frame | Adverse events (AEs) and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 253 days. Treatment emergent AEs are represented for the double-blind period (i.e., starting from randomization to the end of the double-blind period). Total duration of the double-blind period was planned for approximately 36 weeks. | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | ||||||||||
| Arm/Group Title | LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo | |||||
| Arm/Group Description | LIK066 2.5mg once daily | LIK066 10mg once daily | LIK066 50mg once daily | Empagliflozin 25 mg once daily | LIK066 matching placebo and empagliflozin matching placebo | |||||
All Cause Mortality |
||||||||||
| LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
Serious Adverse Events |
||||||||||
| LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 2/15 (13.3%) | 2/16 (12.5%) | 3/30 (10%) | 5/30 (16.7%) | 3/33 (9.1%) | |||||
| Cardiac disorders | ||||||||||
| Angina pectoris | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Atrial fibrillation | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Cardiac failure | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
| Cardiac failure chronic | 0/15 (0%) | 0/16 (0%) | 1/30 (3.3%) | 0/30 (0%) | 0/33 (0%) | |||||
| Cardiac failure congestive | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
| Coronary artery disease | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| General disorders | ||||||||||
| Cardiac death | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Infections and infestations | ||||||||||
| Diarrhoea infectious | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Gastroenteritis | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Wound infection | 0/15 (0%) | 0/16 (0%) | 1/30 (3.3%) | 0/30 (0%) | 0/33 (0%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Hip fracture | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Wound dehiscence | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Nervous system disorders | ||||||||||
| Cerebral vascular occlusion | 0/15 (0%) | 0/16 (0%) | 1/30 (3.3%) | 0/30 (0%) | 0/33 (0%) | |||||
| Reproductive system and breast disorders | ||||||||||
| Benign prostatic hyperplasia | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Respiratory failure | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
Other (Not Including Serious) Adverse Events |
||||||||||
| LIK066 2.5mg | LIK066 10mg | LIK066 50mg | EMPA 25mg | Placebo | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 7/15 (46.7%) | 6/16 (37.5%) | 8/30 (26.7%) | 12/30 (40%) | 9/33 (27.3%) | |||||
| Cardiac disorders | ||||||||||
| Atrial fibrillation | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 2/30 (6.7%) | 1/33 (3%) | |||||
| Gastrointestinal disorders | ||||||||||
| Constipation | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 1/30 (3.3%) | 1/33 (3%) | |||||
| Diarrhoea | 1/15 (6.7%) | 0/16 (0%) | 2/30 (6.7%) | 2/30 (6.7%) | 1/33 (3%) | |||||
| Enteritis | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Flatulence | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Vomiting | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| General disorders | ||||||||||
| Oedema peripheral | 0/15 (0%) | 1/16 (6.3%) | 1/30 (3.3%) | 0/30 (0%) | 1/33 (3%) | |||||
| Pyrexia | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Thirst | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Infections and infestations | ||||||||||
| Breast abscess | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Bronchitis | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 2/33 (6.1%) | |||||
| Genital infection fungal | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Nasopharyngitis | 0/15 (0%) | 1/16 (6.3%) | 1/30 (3.3%) | 1/30 (3.3%) | 2/33 (6.1%) | |||||
| Urinary tract infection | 1/15 (6.7%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Limb injury | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Investigations | ||||||||||
| Heart rate irregular | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Liver function test increased | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Diabetes mellitus inadequate control | 2/15 (13.3%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Fluid retention | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Hyperglycaemia | 0/15 (0%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 2/33 (6.1%) | |||||
| Hypoglycaemia | 1/15 (6.7%) | 2/16 (12.5%) | 2/30 (6.7%) | 3/30 (10%) | 2/33 (6.1%) | |||||
| Hypokalaemia | 0/15 (0%) | 1/16 (6.3%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Arthralgia | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 1/30 (3.3%) | 0/33 (0%) | |||||
| Nervous system disorders | ||||||||||
| Dysaesthesia | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Psychiatric disorders | ||||||||||
| Anxiety | 0/15 (0%) | 1/16 (6.3%) | 1/30 (3.3%) | 0/30 (0%) | 0/33 (0%) | |||||
| Renal and urinary disorders | ||||||||||
| Proteinuria | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Reproductive system and breast disorders | ||||||||||
| Gynaecomastia | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Cough | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 1/33 (3%) | |||||
| Dyspnoea | 0/15 (0%) | 1/16 (6.3%) | 1/30 (3.3%) | 0/30 (0%) | 1/33 (3%) | |||||
| Epistaxis | 1/15 (6.7%) | 0/16 (0%) | 0/30 (0%) | 0/30 (0%) | 0/33 (0%) | |||||
| Vascular disorders | ||||||||||
| Hypotension | 2/15 (13.3%) | 0/16 (0%) | 2/30 (6.7%) | 3/30 (10%) | 1/33 (3%) | |||||
Limitations/Caveats
Due to early discontinuation of the study, the analysis of efficacy was done on the available data (mostly for Epoch 3 (double-blind period 1) only). Because of the small sample sizes the interpretation of the results remained limited.
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
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 (ie, data from all sites) in the clinical trial.
Results Point of Contact
| Name/Title | Study Director |
|---|---|
| Organization | Novartis Pharmaceuticals |
| Phone | 862-778-8300 |
| Novartis.email@novartis.com |
Responsible Party:
Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03152552
Other Study ID Numbers:
- CLIK066B2204
- 2016-003084-19
First Posted:
May 15, 2017
Last Update Posted:
Aug 28, 2019
Last Verified:
Aug 1, 2019