Study of Ruxolitinib in Pancreatic Cancer Patients

Brief summary public domain (ClinicalTrials.gov)

The purpose of this study was to determine whether ruxolitinib added to capecitabine is effective in improving the overall survival of patients with metastatic pancreatic cancer.

Detailed description public domain (ClinicalTrials.gov)

The study consisted of an open-label, safety run-in period that was composed of 1 patient cohort with 9 patients/cohort. This phase of the study determined the safety of the capecitabine/ruxolitinib combination in this patient population. A randomized, double-blind study with two treatment arms was conducted once the safety run-in results from the first part of the study showed that the capecitabine/ruxolitinib combination was safe and additional patients could be treated. All patients have received capecitabine therapy in addition to the ruxolitinib or placebo (Study Drug). Treatment for all patients consisted of repeating 21-day cycles. Capecitabine was self-administered for the first 14 days of each cycle, and the Study Drug was self-administered during the entire 21-day cycle. Treatment cycles continued as long as the regimen was tolerated and the patient did not meet any of the discontinuation criteria. In the event of disease progression, capecitabine therapy was discontinued but the Study Drug could continue to be administered. Subjects who discontinued treatment with the Study Drug continued to be followed to obtain information regarding subsequent treatment regimens and survival.

Posted results (ClinicalTrials.gov registry) · primary outcome public domain (ClinicalTrials.gov)

Overall Survival [Primary analysis includes study data from the start of the study (first dose for that subject) until the death of the subject (up to 8 months).]. Median, days (95% Confidence Interval). Ruxolitinib (n=64): 136.5 (95.0 to 196.0); Placebo (n=63): 129.5 (71.0 to 179.0). Analysis, Ruxolitinib vs Placebo: Hazard Ratio (HR) 0.733 (95% CI 0.506 to 1.061); p=0.0494; Log Rank; superiority or other. Analysis, Ruxolitinib vs Placebo: Hazard Ratio (HR) 0.502 (95% CI 0.281 to 0.888); p=0.0081; Log Rank; superiority or other.

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Progression-Free Survival (PFS) [Analysis includes study data from the start of the study (first dose for that subject) until death or PD, whichever was earlier up to 8 months.]. Median, days (95% Confidence Interval). Ruxolitinib (n=64): 51.0 (42.0 to 84.0); Placebo (n=63): 46.0 (41.0 to 70.0). Analysis, Ruxolitinib vs Placebo: Efron approximation of hazard ratio 0.750 (95% CI 0.513 to 1.094); p=0.1340; Cox proportional hazards model; superiority or other.

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Objective Response Rate [Measured every 4 weeks for duration of study treatment (up to 8 months)]. Number, percentage of participants. Overall response: Ruxolitinib (n=64): 7.8; Placebo (n=63): 1.6. Complete response: Ruxolitinib (n=64): 1.6; Placebo (n=63): 0.0. Partial response: Ruxolitinib (n=64): 6.3; Placebo (n=63): 1.6.

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Durable Response Rate [Measured every 4 weeks until death or PD, whichever was earlier (up to 8 months)]. Number, percentage of participants. Ruxolitinib (n=64): 7.8; Placebo (n=63): 0.0. Analysis, Ruxolitinib vs Placebo: p=0.0236; Pearson's chi-square test; superiority or other.

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Summary of Clinical Benefit [Measured every 4 weeks until death or PD, whichever was earlier (up to 8 months)]. Number, percentage of participants. Subjects with clinical benefit: Ruxolitinib (n=64): 12.5; Placebo (n=63): 1.6. Pain intensity - Improved: Ruxolitinib (n=64): 10.9; Placebo (n=63): 1.6. Analgesic use - Improved: Ruxolitinib (n=64): 4.7; Placebo (n=63): 0.0. Karnofsky performance status - Improved: Ruxolitinib (n=64): 3.1; Placebo (n=63): 0.0. Body weight - Improved: Ruxolitinib (n=64): 3.1; Placebo (n=63): 0.0.