Clinical trial NCT02634723: Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A
Official title: Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2015-12-18
Enrollment: 21
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Previously untreated patients (PUPs) in China with moderate to severe hemophilia A.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of high titer, low titer and transient Factor VIII (FVIII) inhibitors developed
timeFrame: Throughout the study period of 6 months
measure: Number and type of other serious and non-serious adverse events (AEs)
timeFrame: Throughout the study period of 6 months
Secondary outcomes:
measure: Number and type of bleeding episodes treated with ADVATE
timeFrame: Throughout the study period of 6 months
measure: Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE
timeFrame: Throughout the study period of 6 months
measure: Annualized bleeding rate (ABR) of participants treated prophylactically and on-demand with ADVATE
timeFrame: Throughout the study period of 6 months
measure: Annualized joint bleeding rate (AJBR) of participants treated prophylactically and on-demand with ADVATE
timeFrame: Throughout the study period of 6 months
measure: Total number of exposure days (EDs) to ADVATE
timeFrame: Throughout the study period of 6 months
measure: Total units of ADVATE administered
timeFrame: Throughout the study period of 6 months
measure: Total number of exposure days (EDs) to all Factor VIII (FVIII) products
timeFrame: Throughout the study period of 6 months
measure: Total units of all Factor VIII (FVIII) products administered
timeFrame: Throughout the study period of 6 months
Other outcomes:
Locations:
city: Harbin
country: China
facility: Institute of Hematology and Oncology, Harbin The First Hospital
state: Heilongjiang
city: Zhengzhou
country: China
facility: Henan Provincial People's Hospital
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology
state: Hubei
city: Nanjing
country: China
facility: Nanjing Children's Hospital Affiliated to Nanjing Medical University
state: Jiangsu
city: Jinan
country: China
facility: Shandong Blood Center
state: Shandong
city: Hangzhou
country: China
facility: Zhejiang Provincial Hospital of TCM/The First Affiliated Hospital of Zhejiang Chinese Medical University
state: Zhejiang
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: