Clinical trial NCT02170402: China ADVATE PTP Study
Official title: Study to Evaluate Efficacy and Safety of ADVATE in the Treatment of Previously Treated Patients With Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2014-06-23
Enrollment: 82
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)


Eligibility criteria: Main Inclusion Criteria:

* Ethnic Chinese
* is of any age
* has a documented diagnosis of severe or moderately severe hemophilia A (congenital FVIII deficiency: baseline Factor VIII (FVIII) ≤ 2%)
* has documented and verified \>50 exposure days (EDs) to FVIII (recombinant or plasma derived)
* is receiving on-demand treatment with FVIII at the time of enrolment in this study
* has negative history of inhibitor development
* is HIV negative or HIV positive with stable disease and CD4+ count ≥ 200 cells per mm\^3
* is negative for Hepatitis C virus (HCV); Or participant is HCV positive with chronic stable hepatitis as assessed by investigator

Main Exclusion Criteria:

* has prior history of hypersensitivity or anaphylaxis associated with receipt of FVIII
* is diagnosed with other bleeding disorder(s) other than hemophilia A, including but not limited to thrombocytopenia (platelet count \< 100000 /mL)
* has been exposed to an investigational product (IP) within 30 days prior to the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during participation in the study
* is planned, or likely to have surgery during the study period
* has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator
* has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels \> 5 times the upper limit of normal)
* has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent \& persistent international normalized ratio (INR) \>1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
* is a family member of the investigator or site staff

Primary outcomes:
measure: Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment
timeFrame: 12 months
Secondary outcomes:
measure: Number of units per kg body weight of ADVATE required to resolve a bleeding episode (BE)
timeFrame: 12 months
measure: Number of infusions of ADVATE required to resolve a bleeding episode (BE)
timeFrame: 12 months
measure: Overall evaluation of efficacy on a four-point scale (Excellent-Good-Fair-Poor)
timeFrame: 12 months
measure: Annualized bleeding episode rates (ABR) according to bleed type and bleed etiology summarized by treatment regimen
timeFrame: 12 months
measure: Inhibitor incidence
timeFrame: 13 months
measure: Adverse events according to relatedness, seriousness, and severity
timeFrame: 13 months
measure: Area under the plasma concentration/time curve from time 0 to infinity
timeFrame: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion
measure: Mean Residence Time (MRT)
timeFrame: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion
measure: Clearance (CL)
timeFrame: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion
measure: Incremental Recovery (IR) at Cmax
timeFrame: Within 30 minutes prior to the start of the infusion, and within 1 hour post-infusion
measure: Elimination phase half-life
timeFrame: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion
measure: Volume of distribution at steady state (Vss)
timeFrame: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion
Other outcomes:
Locations:
city: Dongcheng
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
state: Fujian
city: Cangzhou
country: China
facility: Cangzhou Central Hospital
state: Hebei
city: Wuhan
country: China
facility: Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech/ Wuhan Union Hospital
state: Hubei
city: Wuhan
country: China
facility: Tongji Hospital of Tongji Medical College of Hongzhong Science and Techology University
state: Hubei
city: Changsha
country: China
facility: Xiangya Hospital Central South University
state: Hunan
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
state: Jiangsu
city: Hangzhou
country: China
facility: The First Affiliated Hospital of College of Medicine, Zhengjiang University
state: Zhejiang
city: Beijing
country: China
facility: Beijing Children's Hospital Affiliated to Capital University of Medical Sciences
city: Shanghai
country: China
facility: Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
city: Tianjin
country: China
facility: Hospital of Blood Disease, Chinese Academy of Medical Sciences
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: