Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A

Completed

• Conditions: Hemophilia A

• Registration Number: NCT01790828
• Lead Sponsor: Pfizer

Brief Summary

This study is to describe the safety and efficacy of Xyntha® during the usual care setting.

Detailed Description

non probability sample

Study Timeline

• Study First Submitted: 2013-02-11
• Study First Submitted QC: 2013-02-11
• Study First Posted: 2013-02-13
• Study Start: 2014-02
• Primary Completion: 2014-08
• Study Completion: 2014-08
• Status Verified: 2015-07
• Results First Submitted: 2015-07-21
• Results First Submitted QC: 2015-07-21
• Last Update Submitted: 2015-07-21
• Results First Posted: 2015-08-17
• Last Update Posted: 2015-08-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

• Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
• Pediatric and adult patients who have been treated with Xyntha for Hemophilia A from first approved date by KFDA, or who are planned to be newly prescribed Xyntha.

Exclusion Criteria

• Patients with a known history of hypersensitivity to original or reformulated Xyntha or any component of the product.
• Patients with a known history of hypersensitivity to hamster protein.
• Patients participating in an interventional trial of any investigational drug or device.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percentage of Participants by Family History of Factor VIII Inhibitor	4 years

Secondary Outcome Measures

Name	Time	Method
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants)	4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age	4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age	4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants)	4 years	Response categories were excellent, good, moderate, or no response.
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age	4 years	Response categories were excellent, good, moderate, or no response.
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age	4 years	Response categories were excellent, good, moderate, and no response.
Number of Participants With LETE Bleeds Within 48 Hours of a Preventive/Prophylaxis Dose of Xyntha	4 years	Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleed wtihin 48 hours of prophylaxis infusion.
Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants)	4 years
Annualized Bleeding Rates During Prophylaxis	4 years	Annualized bleeding rate defined as total number of breakthrough bleeds within 48 hours (for prophylaxis purpose) divided by (/) \[(total period of date of bleeding)/365.25)\]
Average Infusion Dose During Prophylaxis in Participants <18 Years of Age	4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age	4 years
Number of Participants With Less-Than-Expected Therapeutic Effect (LETE) for On-Demand Therapy	4 years	Less than expected therapeutic effect was defined as a 'no response' rating after each of two successive infusions less than or equl to (≤) 24 hours of on-demand therapy.
Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants)	4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age	4 years
Percentage of Participants Experiencing Hemorrhages During Prophylaxis	4 years
Average Infusion Dose During Prophylaxis (All Participants)	4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age	4 years
Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age	4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age	4 years

Trial Locations

Locations (4)

Yonsei Rehabilitation Clinic; 🇰🇷 Guro-gu, Seoul, Korea, Republic of

Pusan National University Hospital; 🇰🇷 Busan, Korea, Republic of

Kim Hugh Chul Internal Medicine Clinic; 🇰🇷 Songpa-gu, Seoul, Korea, Republic of

Kyung Hee University Hospital at Gangdong; 🇰🇷 Seoul, Korea, Republic of