Brief Title
China ADVATE PTP Study
Official Title
Study to Evaluate Efficacy and Safety of ADVATE in the Treatment of Previously Treated Patients With Hemophilia A
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)
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment
Secondary Outcome
Number of units per kg body weight of ADVATE required to resolve a bleeding episode (BE)
Condition
Hemophilia A
Intervention
Octocog alfa (recombinant human coagulation factor VIII)
Study Arms / Comparison Groups
Previously Treated Patients (PTPs)
Description: PTPs will participate sequentially with: Part 1: Pharmacokinetic parameters of ADVATE measured in subset of 24 participants, consisting of: 12 adults (>12 years of age) 12 children (≤12 years of age) Part 2: On-demand treatment with ADVATE for 6 months Part 3: Prophylaxis regimen with ADVATE for 6 months
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
82
Start Date
June 26, 2014
Completion Date
May 31, 2016
Primary Completion Date
May 31, 2016
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
Gender
Male
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT02170402
Organization ID
061301
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Takeda
Verification Date
April 2021