China ADVATE PTP Study

Baxalta

Status and phase: Completed; Phase 4

Conditions: Hemophilia A

Treatments: Biological: Octocog alfa (recombinant human coagulation factor VIII)

Study type: Interventional

Funder types: Industry

Identifiers

NCT02170402

061301

Details and patient eligibility

About

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

Enrollment: 82 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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82 participants in 1 patient group

Previously Treated Patients (PTPs)

Experimental group

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

Treatment:

Biological: Octocog alfa (recombinant human coagulation factor VIII)