Trial Outcomes & Findings for Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B (NCT NCT04286412)

The study was conducted in India from 10 Feb 2020 to 24 Sep 2020. A total of 25 participants were enrolled.

In this study, participants aged greater than or equal to 12 years to less than or equal to 65 years with congenital moderately-severe to severe hemophilia B (factor IX activity less than or equal to 2 percent) who had at least 50 exposure days (EDs) (ED was defined as a 24 hours period in which the participant was administered any FIX-containing product) to FIX-containing products were enrolled.

Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.

An adverse events (AE) is any untoward medical occurrence in clinical investigation participant administered a product or medical device; event need not necessarily to have a causal relationship with treatment or usage. SAEs: an AE resulting in any of following outcomes/deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. MIEs: any confirmed FIX inhibitor development (titer greater than or equal to 0.6 BU/mL confirmed by central laboratory testing), thrombotic events (any event associated with the formation of a blood clot including catheter-associated thrombi and thrombotic complications) or hypersensitivity reactions (hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgment).

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are events between first dose of study drug and up to 116 days that were absent before treatment or that worsened relative to pretreatment state.

ABR: number of bleeding episodes per year. ABR for each participant = number of bleeds during treatment interval duration (TID)/(TID/365.25). Number of bleeds for each participant for ABR included all new bleeds (with unique start date and time) requiring treatment with nonacog alfa during TID. TID=date of Visit 4 (3 to 10 days after last dose) - date of Visit 2 (Day 1) +1. For participants who had Visit 4 beyond 3 (+7) days after final dose due to COVID-19 pandemic, date of final dose was used in place of date of Visit 4. For discontinued participants, date of final dose/last study visit date (whichever occurred later) was used to replace Visit 4.

The TFC per participant was the sum of the total amount (in international units \[IU\]) infused for each infusion for each participant. Annualized TFC of nonacog alfa was derived for each participant by using the following formula; Annualized TFC = (TFC / treatment interval duration)\*365.25. Treatment interval duration was calculated as the number of days beginning on Visit 2 ("Day 1", provided an infusion was given) up to Visit 4 (any 1 day between Day 52 to Day 60).

The total amount in international units (IU) per kilogram infused for each infusion recorded were summed to calculate the total factor consumption for each participant. Annualized TFC = (TFC / treatment interval duration)\*365.25. Treatment interval duration was calculated as Date of Visit 4 - Date of Visit 2 +1. If Date of Visit 4 - Last dosing date was \> 10 days then Date of Visit 4 was replaced with Last dosing date. If Date of Visit 4 was missing (due to discontinuation or any other reason), then Date of Visit 4 was replaced with last dosing date or last subject visit date, whichever was greater. International unit per kilogram= IU/kg.

Number of nonacog alfa infusions used to treat each bleed in participants was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). On demand treatment was defined as treatment used to treat a bleeding episode.