Summary ID# 13614. Clinical Study Summary: Study F3Z-JE-PV06



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CT Registry ID# Page 1 Summary ID# 13614 Clinical Study Summary: Study F3Z-JE-PV06 INSIGHTS; INSulin-changing study Intending to Gain patients insights into insulin treatment with patient-reported Health outcomes in actual clinical TreatmentS Date summary approved by Lilly:

CT Registry ID# Page 2 Title of Study: INSIGHTS; INSulin-changing study Intending to Gain patients insights into insulin treatment with patient-reported Health outcomes in actual clinical TreatmentS. Investigator(s): This a prospective, non-safety observational study included 58 principal investigator(s). Study Center(s): This study was conducted at 58 study center(s) in one country Length of Study: Phase of Development: Phase4 Date of first patient enrolled: 22 Jan 2010 Date of last patient completed entire study: 30 Nov 2010 Objectives: Primary objective to assess changes in quality of life (QOL) associated with insulin regimen changes in patients with type 2 diabetes mellitus (T2DM). Secondary objective to determine the reasons for and patterns of insulin regimen changes and the effects on glycaemic control. Study Design: A prospective, non-safety observational study (12weeks). Number of Patients/Subjects: Planned: 700 patients Registered: 674 patients Evaluated: 625 patients Main Criteria for Inclusion: Patients with T2DM aged 20 years who planned to change one or more aspect of their insulin regimen (type of insulin, injecting device, and / or number of injections per day) during the registration period Test Product/Study Drug, Dose, and Mode of Administration: Patients individual insulin regimens during the study were determined by their treating doctors, and each insulin preparation was to be administered according to the recommended dose and frequency. Reference Therapy/Comparator, Dose, and Mode of Administration: Not applicable Duration of Treatment: 12 weeks Variables: Primary endpoint The change from baseline in the total score of ITR-QOL at Week 12 will be determined with its 95% confidence interval. Secondary endpoint reasons for changes in insulin therapy mean changes from baseline in HbA1c and DTSQ scores pattern of changes in insulin therapy

CT Registry ID# Page 3 Evaluation Methods: Statistical: The software package nquery Advisor (Version 5.15.1, Statistical Solutions Ltd, Cork, Ireland) was used to calculate planned sample size. The standard deviation (SD) of the change in the total ITR-QOL score was assumed to be 12.0, based on previous results. To estimate the change from baseline in the ITR-QOL total score with sufficient precision (95% CI for the estimated mean change of 2.0), 554 patients were needed. Allowing for 20% dropout, the study aimed to include approximately 700 patients. Summary statistics (mean, SD) were calculated for all continuous variables (eg, age, weight, ITR-QOL scores, HbA1c levels). A one-sample t test was used to compare baseline and Week 12 values within patient subgroups, and a two-sample t test was used to compare changes from baseline between patient subgroups. A multiple linear regression model was used to assess the relationship between independent variables (age, sex, BMI, baseline HbA1c level, duration of diabetes, number of insulin injections, type of insulin, and device) and the change from baseline in plasma HbA1c level. A backward, step-down method was used to further assess variables with P < 0.05 in the full regression model. Missing data were not imputed. All statistical analyses were conducted using SAS, Version 9.1.3 (SAS, Cary, NC) and P values 0.05 were considered significant. Summary: Patient disposition and baseline characteristics A total of 674 patients were registered, of whom 625 were evaluated. The most common reason for exclusion of registered patients from evaluation was protocol violation. The most common reason for discontinuation during the study was a subsequent change in insulin regimen. The baseline characteristics of the patients were typical of Japanese patients with T2DM (Table 1). Table 1. Patient Characteristics at Baseline. Variable Value N = 625 Age, years (mean ± SD) 63.3 ± 12.0 Sex, n (%) Men 339 (54.2%) Women 286 (45.8%) Height, cm (mean ± SD) 160.73 ± 9.47 Mean (SD) Weight, kg (mean ± SD) 63.59 ± 13.82 Body mass index, kg/m 2 (mean ± SD) 24.52 ± 4.32 Duration of diabetes, years (mean ± SD) 16.15 ± 8.91 Duration of insulin treatment, years (mean ± SD) 7.17 ± 5.53 Concomitant oral antidiabetic medication, n (%) None 259 (41.4%) At least one type a 366 (58.6%) Biguanide 189 (30.2%) α-glucoside inhibitor 161 (25.8%) Sulfonylurea 157 (25.1%) Thiazolidinedione 71 (11.4%) Glinide 18 (2.9%) Complications of diabetes, n (%) Present 540 (86.4%) Absent 84 (13.4%)

CT Registry ID# Page 4 Variable Value N = 625 Unknown 1 (0.2%) Type of complication, n (%) Hypertension 334 (53.4%) Hyperlipidemia 320 (51.2%) Diabetic retinopathy 287 (45.9%) Diabetic neuropathy 238 (38.1%) Diabetic nephropathy 220 (35.2%) Quality of life Quality of life did not worsen during the 12-week study. There was no statistically significant change in the ITR- QOL total score from baseline to Week 12 (Table 2). Similarly, there was no significant change in the ITR-QOL total score when results were stratified by the type of change (insulin type, injection device, or number of injections). There was a small, but statistically significant, increase in the daily activities subscale score; no other subscale score changed significantly. There was a small, but statistically significant, increase in the daily activities subscale score; no other subscale score changed significantly. The perceived frequency of hyperglycaemia DTSQ subscale score (mean ± SD) decreased significantly (P ; n = 573) from 3.4 ± 1.6 at baseline to 3.0 ± 1.7 at Week 12, in accordance with improvement of glycaemic control. There were no statistically significant changes in the mean treatment satisfaction (baseline: 24.8 ± 6.4; Week 12: 25.4 ± 6.4; n = 552) or perceived frequency of hypoglycaemia (baseline: 1.7 ± 1.5; Week 12: 1.8 ± 1.5; n = 571) DTSQ subscale scores. Table 2. Insulin Therapy-related Quality of Life Subscale and Total Scores During the 12-Week Study Subscale (range of possible scores) Baseline N = 625 Social activities (5-25 points) 22.0 (3.7) n = 609 Physical functioning (4-20 points) 17.3 (2.5) n = 612 Daily activities (3-15 points) 12.7 (2.3) n = 614 Feeling about insulin treatment (11-55 points) 44.6 (9.0) n = 593 Total score (23-115 points) 97.0 (15.2) n = 575 a One-sample t test. Abbreviation: SD = standard deviation Score Mean (SD) Week 12 N = 611 22.2 (3.7) n = 583 17.4 (2.6) n = 583 12.9 (2.3) n = 578 45.1 (9.3) n = 591 97.8 (15.6) n = 560 Difference 0.12 (2.99) n = 567 0.04 (2.17) n = 571 0.18 (2.08) n = 568 0.54 (7.06) n = 562 0.86 (11.25) n = 518 P Value a 0.341 0.672 0.038 0.073 0.082 Reasons for change in insulin regimen Overall, the most common reason for changing insulin regimen was poorly controlled glycaemia. This was the most common doctor-reported reason and the second most common patient-reported reason. Among patients, the most common reason for change was recommendation from the doctor, which may have been related to poorly

CT Registry ID# Page 5 controlled glycaemia, although this cannot be determined from the questionnaires. Change in insulin product was the second most common doctor-reported reason and the third most common patient-reported reason. Insulin types and injection times The three categories of insulin type were used with similar prevalence both before and after the change in insulin therapy. Rapid-acting insulin was most commonly injected before breakfast, before lunch, and before dinner. Biphasic insulin was most commonly injected before breakfast and before dinner. Long-acting insulin was most commonly injected before breakfast, before dinner, or at bedtime. Combinations of insulin types Most patients used one or two types of insulin both before and after the change in insulin regimen. Biphasic insulin only (ie, without an additional insulin type) was the most common insulin used both before and after the change in regimen. The change in insulin regimen was associated with a decrease in the proportion of patients using one type of insulin (from 64.5% to 55.4%) and an increase in the proportion using two types of insulin (from 34.4% to 43.0%). Injection devices The proportion of patients using each type of injection device did not change substantially after the change in insulin regimen. Disposable devices were the most common devices used both before (n = 471 patients; 75.4%) and after (n = 486 patients; 77.8%) the change in regimen. Cartridges (before: n = 127 patients, 20.3%; after: n = 94 patients, 15.0%) and syringes (before: n = 3 patients, 0.5%; after: n = 2 patients, 0.3%) were also used. A small number of patients used both disposable devices and cartridges (before: n = 24 patients, 3.8%; after: n = 43 patients, 6.9%). Number of injections Most patients injected insulin two or three times a day both before and after the change in insulin regimen. However, the proportion of patients who injected three or four times a day increased after the change in regimen. The distribution of the number of injections per day at Week 12 was similar to the distribution immediately after the change. Total daily insulin dose There was a small, but statistically significant, increase in the total daily dose of insulin (baseline: 30.7 ± 21.8 IU; Week 12: 31.5 ± 20.1 IU; change = +0.8 ± 9.0 IU; n = 620; P = 0.023) during the study, which was not considered clinically significant. Patient-reported treatment adherence

CT Registry ID# Page 6 The change in insulin regimen was not associated with any apparent change in patient-reported treatment adherence. Most patients reported adhering to treatment always or often at all injection times both before and after the change in insulin regimen. Concomitant oral antidiabetic medication The proportion of patients who were taking concomitant oral antidiabetic medication decreased slightly after the change in insulin regimen (before change: n = 366; 58.6%; after change: n = 347; 55.5%) and was maintained at Week 12 (or discontinuation) (n = 348; 55.7%). Overall changes in insulin therapy Nearly a third (188 of 625; 30.1%) of patients changed more than one aspect of their insulin regimen. Most (521 of 625; 83.4%) patients changed the type of insulin as part of the change in insulin regimen. Among patients who changed the number of injections, most (132 of 183; 72.1%) also changed the type of insulin. Plasma HbA1c levels Plasma HbA1c levels decreased significantly (P ) from 8.21 ± 1.87% (n = 618) at baseline to 7.85 ± 1.71% (n = 610) at Week 12 (change = -0.37 ± 0.97%, n = 606). Stratified analysis based on the type of change showed statistically significantly greater decreases in plasma HbA1c levels were observed in patients who changed the type of insulin or the number of injections compared with patients who did not make these changes (Table 3). Table 3. Stratified Analysis of Changes in Plasma Glycated Haemoglobin Levels During the 12-Week Study. HbA1c Mean % (SD) Insulin Therapy Change Baseline Week 12 Difference from Baseline Type of insulin Not changed 8.11 (1.56) n = 135 Changed 8.24 (1.44) n = 483 Type of injection device Not changed 8.24 (1.44) n = 493 Changed 8.11 (1.55) n = 125 7.87 (1.35) n = 132 7.85 (1.29) n = 478 7.85 (1.33) n = 487 7.80 (1.21) n = 123-0.21 (0.80) n = 132-0.41 (1.01) n = 474-0.38 (1.00) n = 483-0.31 (0.86) n = 123 Within Group P Value a 0.003 Number of injections Not changed 8.01 (1.35) 7.77 (1.33) -0.25 (0.84) Difference Between Groups b (95% CI) -0.20 (-0.39, -0.01) 0.07 (-0.12, 0.26) Betwe P 0 0

CT Registry ID# Page 7 HbA1c Mean % (SD) Insulin Therapy Change Baseline Week 12 Difference from Baseline n = 438 n = 431 n = 429 Changed 8.72 (1.62) 8.06 (1.22) -0.66 (1.18) n = 180 n = 179 n = 177 Within Group P Value a Difference Between Groups b (95% CI) -0.42 (-0.58, -0.25) Betwe P <