Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A

Completed

• Conditions: Hemophilia A
• Interventions: Biological: Octocog alfa (recombinant human coagulation factor VIII)

• Registration Number: NCT02634723
• Lead Sponsor: Baxalta now part of Shire

Brief Summary

The purpose of this post-marketing safety study is to evaluate the safety, immunogenicity, and effectiveness of ADVATE in previously untreated patients (PUPs) in China with moderate to severe hemophilia A.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-12-16
• Study First Submitted QC: 2015-12-16
• Study First Posted: 2015-12-18
• Study Start: 2015-12-23
• Primary Completion: 2016-06-30
• Study Completion: 2016-06-30
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-16
• Last Update Posted: 2021-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

1. Previously untreated patient (PUP) in China with moderate to severe hemophilia A.
2. Investigator prescribed ADVATE as the predominant therapeutic agent for the management of hemophilia A
3. Informed consent form from patient and/or legal representative will need to be signed per local regulation

Exclusion Criteria

1. Presence of an inherited or acquired hemostatic defect other than hemophilia A, and any other clinically significant chronic disease
2. Participants who participated in another investigational FVIII drug study, or had participated in any clinical study involving an investigational FVIII drug during the course of the observation period

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Previously Untreated Patients (PUPs)	Octocog alfa (recombinant human coagulation factor VIII)	PUPs in China with Moderate to Severe Hemophilia A

Primary Outcome Measures

Name	Time	Method
Number and type of other serious and non-serious adverse events (AEs)	Throughout the study period of 6 months	Other AEs refers to AEs other than Factor VIII (FVIII) inhibitors
Number of high titer, low titer and transient Factor VIII (FVIII) inhibitors developed	Throughout the study period of 6 months

Secondary Outcome Measures

Name	Time	Method
Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE	Throughout the study period of 6 months
Annualized joint bleeding rate (AJBR) of participants treated prophylactically and on-demand with ADVATE	Throughout the study period of 6 months
Total units of all Factor VIII (FVIII) products administered	Throughout the study period of 6 months
Total number of exposure days (EDs) to ADVATE	Throughout the study period of 6 months
Total units of ADVATE administered	Throughout the study period of 6 months
Number and type of bleeding episodes treated with ADVATE	Throughout the study period of 6 months
Annualized bleeding rate (ABR) of participants treated prophylactically and on-demand with ADVATE	Throughout the study period of 6 months
Total number of exposure days (EDs) to all Factor VIII (FVIII) products	Throughout the study period of 6 months

Trial Locations

Locations (6)

Henan Provincial People's Hospital; 🇨🇳 Zhengzhou, Henan, China

Nanjing Children's Hospital Affiliated to Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China

Zhejiang Provincial Hospital of TCM/The First Affiliated Hospital of Zhejiang Chinese Medical University; 🇨🇳 Hangzhou, Zhejiang, China

Shandong Blood Center; 🇨🇳 Jinan, Shandong, China

Institute of Hematology and Oncology, Harbin The First Hospital; 🇨🇳 Harbin, Heilongjiang, China

Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology; 🇨🇳 Wuhan, Hubei, China