Clinical trial NCT03034044: Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
Official title: POST-MARKETING SURVEILLANCE TO OBSERVE SAFETY AND EFFICACY OF XYNTHA SOLOFUSE PREFILLED SYRINGE
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Factor VIII Deficiency, Congenital
Factor 8 Deficiency, Congenital
Autosomal Hemophilia A
Classic Hemophilia
Hemophilia A, Congenital
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2017-01-27
Enrollment: 106
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population is Hemophilia A (congenital factor VIII deficiency) patients who have been administered the Xyntha Solofuse prefilled syringe (as part of routine treatment at the Korean health care canter which has certified investigators).

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.


Eligibility criteria: -. Inclusion criteria

To be eligible to enroll in this study, the study subjects will have to meet all the following inclusion criteria:

1. Hemophilia A (congenital factor VIII deficiency) patients who have been administered according to the indication of the product 1) Control and prevention of bleeding episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency) 2) This drug does not contain von Willebrand factor and, therefore, is not indicated in von Willebrand's disease
2. Those who have been administered the Xyntha Solofuse prefilled syringe at least once - Exclusion criteria

Patients who satisfy the following criteria are not included in the study according to the local labeling:

1. Patients who have a history of hypersensitivity to the Xyntha Solofuse prefilled syringe or the ingredients of this drug.
2. Patients who have a history of hypersensitivity to hamster proteins.
3. Patients who have bleeding disorders other than hemophilia A.
4. Patients who have a history of FVIII inhibitors, or currently have or are suspected of having FVIII inhibitors. In case inhibitor titers quantified in Bethesda Units in the laboratory test results are within the normal laboratory range or at least 0.6 BU/mL. If laboratory tests cannot be performed, the investigator will determine whether or not inhibitors exist based on the clinical assessment results that show a decrease in efficacy of the replacement of FVIII (e.g. bleeding at least once, if the replacement of anti-bleeding agents is needed to be administered, and if frequency or dosage of replacement FVIII therapy needs to be increased).
5. Use of immunomodulatory therapy. (e.g. intravenous injection of immunoglobulin, use of regular systemic corticosteroids, cyclosporine, and mediators of anti-TNF-α)

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants With Adverse Events (AEs) by Severity
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants Who Discontinued Due to Adverse Events
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants Who Died Due to Adverse Events
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding
timeFrame: From the first administration of Xyntha up to 6 months
measure: Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery
timeFrame: Within 24 hours of on-demand treatment (anytime within the observation period of 6 months)
measure: Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding
timeFrame: Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months)
measure: Number of Infusions Required to Treat Each New Bleeding Episode
timeFrame: From the first administration of Xyntha up to 6 months
measure: Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery
timeFrame: From the first administration of Xyntha up to 6 months
measure: Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding
timeFrame: From the first administration of Xyntha up to 6 months
measure: Percentage of Participants With Bleeding Event
timeFrame: From the first administration of Xyntha up to 6 months
measure: Annualized Bleeding Rates (ABRs)
timeFrame: Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months)
measure: Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy
timeFrame: From the first administration of Xyntha up to 6 months
measure: Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy
timeFrame: From the first administration of Xyntha up to 6 months
measure: Total Factor VIII Consumption
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: