Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe

Completed

Conditions: Factor VIII Deficiency, Congenital
Autosomal Hemophilia A
Hemophilia A, Congenital
Factor 8 Deficiency, Congenital
Classic Hemophilia

Registration Number: NCT03034044

Lead Sponsor: Pfizer

Brief Summary: This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor). The secondary objective is to observe the overall efficacy and safety of the Xyntha Solofuse prefilled syringe including serious adverse events. In this open-label, non-comparative, observational, non-interventional, retrospective and multi-center study, post-marketing surveillance data will be collected retrospectively for up to 6 months from the initial administration day of the Xyntha Solofuse prefilled syringe injected into patients who have been administered the Xyntha Solofuse prefilled syringe.

As specified in the product approval issued by the Ministry of Food and Drug Safety, the study will be conducted for 4 years from the approval date. At least 600 study subjects will be enrolled in this study to meet the MFDS requirements. Although 600 is the assigned number of study subjects, the number of cases will be adjusted considering the actual number of enrolled subjects after the study start day.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 106

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	From the first administration of Xyntha up to 6 months	An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment Emergent Adverse Event (TEAE) was adverse event that started or worsened in severity after first administration of Xyntha Solofuse up to 6 months. AEs included both serious and non-serious adverse event.
Number of Participants Who Discontinued Due to Adverse Events	From the first administration of Xyntha up to 6 months	An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship.
Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy	From the first administration of Xyntha up to 6 months	The average dose of Xyntha per bleeding event according to prophylaxis therapy treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. Prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding).
Number of Participants Who Died Due to Adverse Events	From the first administration of Xyntha up to 6 months	An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship.
Percentage of Participants With Bleeding Event	From the first administration of Xyntha up to 6 months
Number of Participants With Adverse Events (AEs) by Severity	From the first administration of Xyntha up to 6 months	An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs were classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function, and was determined based on investigator's discretion.
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events	From the first administration of Xyntha up to 6 months	Treatment-related AE was any untoward medical occurrence attributed to Xyntha in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Relatedness to treatment was assessed by investigator. AEs included both serious and non-serious AEs.
Number of Infusions Required to Treat Each New Bleeding Episode	From the first administration of Xyntha up to 6 months	It was calculated as total number of injections given throughout the study divided by total number of bleeding events.
Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding	From the first administration of Xyntha up to 6 months	The average dose of Xyntha per bleeding event was calculated as total dose of Xyntha throughout the study (in International Units \[IU\]) divided by total number of bleeding incidence. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding means treatment administered due to spontaneous bleeding or abrasion.
Total Factor VIII Consumption	6 months	Total factor VIII consumption for each participant was calculated by sum of the total amount of Xyntha Solofuse (in IU) infused for each Xyntha Solofuse infusion.
Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery	Within 24 hours of on-demand treatment (anytime within the observation period of 6 months)	LETE for on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment.
Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding	Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months)	LETE for on-demand treatment ((administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment.
Annualized Bleeding Rates (ABRs)	Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months)	Annualized bleeding rate defined as number of bleeds under prophylactic setting (defined as bleeding occurred after 48 hours of prophylactic therapy \[administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding\]) divided by (/) \[(number of prophylactic therapy participants)\*0.5)\]
Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy	From the first administration of Xyntha up to 6 months	Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as: administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding).
Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery	From the first administration of Xyntha up to 6 months	On-demand treatment according to surgery: treatment to increase factor in preparation for surgery. Overall responses to all injection of Xyntha Solofuse prefilled syringe used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate and 4=no response, higher score = better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement starting after 8 hours following infusion, with \>=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion/condition worsens
Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding	From the first administration of Xyntha up to 6 months	On-demand treatment according to bleeding: treatment administered for spontaneous bleeding/abrasion; not requiring surgery. Overall responses to all injection of Xyntha used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate,4=no response, higher score=better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement after 8 hours following infusion, with \>=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion, or condition worsens.
Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery	From the first administration of Xyntha up to 6 months	The average dose of Xyntha per bleeding event according to surgery in on-demand treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery means treatment to increase factor in preparation for surgery.