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Efficacy and Safety of 3 Different IV Doses of Palonosetron for the Prevention of PONV in the Outpatient (Study 1) and Inpatient (Study 2) Settings Keith Candiotti, MD Departments of Anesthesiology, Perioperative Medicine and Pain Management University of Miami Miami, Florida American Society of Anesthesiologists Annual Meeting October 20, 2008 Study 1 (Outpatients—US): Candiotti KA, et al. A randomized, double-blind study to evaluate the efficacy and safety of three different doses of palonosetron versus placebo for preventing postoperative nausea and vomiting. Anesth Analg. 2008;107:445-451. Study 2 (Inpatients—Europe): Kovac A, et al. A randomized, double-blind study to evaluate the efficacy and safety of three different doses of palonosetron versus placebo in preventing postoperative nausea and vomiting over a 72-hour period. Anesth Analg. 2008;107:439-444. 1 Palonosetron PONV Trials • Study funded by MGI/Eisai • Study presented on behalf of all investigators involved in both trials. 2 Phase 3 Study Designs Two Randomized, Stratified, Double-blind, Parallel-group, Balanced, Placebo-controlled Multicenter Studies Patients Study 1: Outpatients (US) Study 2: Inpatients (Europe) M/F pts undergoing elective laparoscopic abdominal or gynecological surgery Female pts undergoing elective gynecological or breast surgery Randomization Stratification Placebo vs PALO dosage Interactive voice response system Hx of PONV/motion sickness Non-smoking status Gender Placebo (1) n = 135 (2) n = 136 Hx of PONV/motion sickness Non-smoking status Type of surgery PALO 0.025 mg/kg IV PALO 0.050 mg/kg IV PALO 0.075 mg/kg IV (1) n = 136 (2) n = 136 (1) n = 137 (2) n = 137 (1) n = 138 (2) n = 135 Patient Demographics Study 1: Outpatients (US) Study 2: Inpatients (Europe) Palonosetron (IV) Palonosetron (IV) Placebo 0.025 mg/kg 0.050 mg/kg 0.075 mg/kg Placebo 0.025 mg/kg 0.050 mg/kg 0.075 mg/kg (n=135) (n=136) (n=137) (n=138) (n=136) (n=136) (n=137) (n=135) Gender (female) 96 96 96 96 100 100 100 100 Mean age (years) 37 39 37 38 50 48 48 50 Hx of PONV/motion sickness 64 63 66 66 46 47 47 47 Smoking status (nonsmoker) 86 85 85 85 85 86 85 84 Alcohol consumption (no) 50 54 58 57 38 33 35 33 Gynecologic laparoscopy 78 75 77 69 -- -- -- -- Abdominal laparoscopy 22 25 23 31 -- -- -- -- Gynecologic surgery -- -- -- -- 84 81 84 84 Breast surgery -- -- -- -- 16 19 16 16 ASA status I 42 52 50 52 36 48 45 50 ASA status II 53 43 45 45 61 49 52 48 ASA status III 5 5 4 3 3 3 3 2 4 Stratification: PONV Risk Factors (% of Patients) Placebo PALO 0.025 mg IV PALO 0.050 mg IV PALO 0.075 mg IV Study 1: Outpatients (US) Male + hx PONV + non-smoker 4 4 4 4 Female + hx PONV + non-smoker 47 44 47 46 Female + hx PONV + smoker 13 15 15 15 Female + no hx PONV + non-smoker 36 37 34 34 Hx no PONV/motion sickness + non-smoker 54 53 53 53 Hx PONV/motion sickness + non-smoker 32 33 33 31 Hx PONV/motion sickness + smoker 15 14 15 16 Study 2: Inpatients (Europe) There were no statistically significant differences in PONV risk factors in patients across all treatment groups. 5 Anesthesia • Premeds – Midazolam 1-2 mg IV or fentanyl 50-100 g IV as needed • Induction – Propofol 2.0-2.5 mg/kg IV or thiopental 2.5-5.0 mg/kg IV; methohexital 1.5-3.0 mg/kg IV – Succinylcholine 1-1.5 mg/kg IV; rocuronium 0.4-0.6 mg/kg IV, cisatracuronium 0.15-0.3 mg/kg IV, vecuronium 0.05-0.1 mg/kg IV, or mivacurium 0.015-0.25 mg/kg • Maintenance – N2O 50-70% end tidal concentration, O2 30-50% end tidal concentration – Rocuronium, cisatracuronium or vecuronium titrated as needed for muscle relaxation • Inhalation agent – Isoflurane 0.4-3%, desflurane 2-6% or sevoflurane 1-3% end tidal concentration, titrated as needed • Intraoperative opioid – Fentanyl 2-10 g/kg IV or sufentanil 0.2-0.6 g/kg, titrated as needed • Muscle relaxant reversal – Neostigmine <or= 2.5 mg IV and glycopyrrolate 0.4-0.8 mg IV as per usual clinical practice 6 Study Endpoints Primary endpoint for both studies • Complete response* (CR) at 0-24 h and 24-72 h (no emetic episodes and no rescue medication) Secondary endpoints for both studies† • • • • • • • CR at additional time intervals Complete control (CR plus no more than mild nausea) Percentage of pts experiencing emesis Number of emetic episodes Severity of nausea Time to treatment failure Patient functional interference (Study 1 only) * P value adjusted for 3-dose comparison vs placebo [P=0.05/3=0.0166] † Measured at 2, 6, 24, 48, and 72 h (all tested at P<0.05) 7 Complete Response (No Emesis, No Use of Rescue Medication) Study 2 Study 1 100 90 * % of Patients 80 70 70 * 60 * * 43 50 40 30 33 39 41 44 † † 47 49 26 56 24 32 37 39 46 47 52 56 † 61 44 45 36 52 36 20 10 0 0-24 h 24-72 h 0-72 h Primary endpoints * Statistically significant at P <0.0166 (for primary analyses); analysis by logistic regression. at P <0.05 (for secondary analyses); analysis by logistic regression. †Statistically significant 0-24 h 24-72 h 0-72 h Primary endpoints 8 Percentage of Patients With No Treatment Failure Study 2: Inpatients (Europe) % of Patients With No Treatment Failure Study 1: Outpatients (US) 100 100 80 80 60 60 PALO 0.075 mg 40 Placebo PALO 0.075 mg 40 Placebo 20 20 0 0 0 20 40 60 80 0 20 40 60 Hours Hours P = 0.0185 for palonosetron 0.075 mg vs placebo. P = 0.0035 for palonosetron 0.075 mg vs placebo. Time to treatment failure: time to first emetic episode and/or to first use of rescue meds. Patients who did not have treatment failure were censored at 72 hours. 80 9 Nausea Severity Study 1 * 100 % of Patients Evaluable for Nausea Study 2 * * * 80 Severe Moderate Mild None 60 40 20 0 Placebo PALO 0.075 mg 0-24 h Placebo PALO 0.075 mg 0-72 h * Statistically significant vs placebo at P<0.05 (Cochran-Mantel-Haenszel). Placebo PALO 0.075 mg 0-24 h Placebo PALO 0.075 mg 0-72 h 10 Percentage of Patients Without Functional Interference* (Study 1 Only) Placebo % of Patients Without Interference 100 PALO 0.075 mg † 80 60 † 57 64 73 59 65 † 73 66 62 57 44 40 20 0 Appetite Sleep Physical activities Social life Enjoyment of life 0-24 h * Functional interference due to nausea and vomiting was measured utilizing a Modified Osoba Nausea and Emesis Module. Measurements were based on a 4-point Likert scale: 1 = not at all; 2 = a little; 3 = quite a bit; 4 = very much. The percentages provided above represent those patients with a score of 1 on any individual subscale. † P<0.05 (Mann-Whitney test for pair-wise comparisons of palonosetron vs placebo). 11 Most Frequent Treatment-related Adverse Events Study 1: Outpatients (US) Adverse Events: n, % Placebo (n=137) PALO 0.025 mg IV (n=136) PALO 0.050 mg IV (n=138) PALO 0.075 mg IV (n=136) Total treatment-related AEs 13 (10%) 12 (9%) 15 (11%) 9 (7%) Headache 6 (4%) 5 (4%) 9 (7%) 4 (3%) Constipation 4 (3%) 2 (2%) 6 (4%) 4 (3%) Study 2: Inpatients (Europe) Adverse Events: n, % (n=136) (n=136) (n=137) (n=135) Total treatment-related AEs 45 (27%) 48 (29%) 49 (29%) 48 (29%) Bradycardia 15 (9%) 16 (10%) 14 (8%) 13 (8%) ECG QTc prolonged 11 (7%) 10 (6%) 14 (8%) 15 (9%) There were no statistically significant differences among treatment groups. 12 Changes in QTc Placebo (n=137) PALO 0.025 mg IV (n=136) PALO 0.050 mg IV (n=138) PALO 0.075 mg IV (n=136) 15 minutes 18 15 13 16 6 hours 9 10 11 11 (n=168) (n=168) (n=169) (n=168) 15 minutes 20 24 22 21 6 hours 10 11 13 11 Study 1: Outpatients (US) Mean change from baseline in QTc by Fridericia, msec Study 2: Inpatients (Europe) Mean change from baseline in QTc by Fridericia, msec ECGs: In triplicate at baseline (screening) and as a single recording at 15 minutes and at 3-6 hours (Study 1) and 6 hours (Study 2) post-study drug administration. Independent blinded cardiologist reading with manual QT interval measurement. Results: At 15 minutes post-dose, QT prolongation was consistent across all treatment groups. At 3 to 6 hours post-dose (Study 1) and at 6 hours post-dose (Study 2), QTc intervals remained slightly increased from baseline values; however, they tended to normalize compared with the 15minute results. 13 Overall Summary • Dose-response trend observed with increasing doses of PALO • A single IV dose of PALO (0.075 mg) : • was effective in reducing PONV in both the inpatient and outpatient settings • was superior to placebo (0-24 hours) for the primary endpoint (CR) • reduced the severity of nausea compared with placebo • Adverse events: no dose relationship, no differences vs placebo, consistent with those expected for the 5-HT3 receptor antagonist class • These benefits may: • distinguish PALO as unique among the 5-HT3 receptor antagonists • address important unmet needs, including nausea control 14