A Phase 2b, Randomized, Double-blind, Placebo-Controlled Multicenter Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of Presatovir in Hematopoietic Cell Transplant Recipients with Respiratory Syncytial Virus (RSV) Infection of the Lower Respiratory Tract

Marty, Francisco M; Chemaly, Roy F; Mullane, Kathleen M; Lee, Dong-Gun; Hirsch, Hans H; Small, Catherine B; Bergeron, Anne; Shoham, Shmuel; Ljungman, Per; Waghmare, Alpana; Blanchard, Elodie; Kim, Yae-Jean; McKevitt, Matt; Porter, Danielle P; Jordan, Robert; Guo, Ying; German, Polina; Boeckh, Michael; Watkins, Timothy R; Chien, Jason W; Dadwal, Sanjeet S

The primary analysis tested superiority of presatovir vs placebo using parametric analysis of covariance using baseline viral load and randomization stratification factors as covariates with a 2-sided α of 0.05 (Supplemental methods). Number of supplemental oxygen-free days was analyzed using a negative binomial model with stratification factors as covariates and an offset parameter to account for on-study duration. Patients who died prior to day 28 or received supplemental oxygen on all days of the study period were assigned a value of 0 supplemental oxygen-free days. The proportion of patients developing respiratory failure of any cause requiring invasive or noninvasive mechanical ventilation through day 28 and allcause mortality through day 28 were analyzed using Cochran-Mantel-Haenszel tests adjusting for the stratification factors at the 2-sided 0.05-level, with 2-sided 95% exact confidence interval (CI) based on the Clopper-Pearson method for each treatment group. Where number of events was small, Fisher exact test was used.