Pharmacokinetics, Efficacy and Safety Study of IMMUNATE SD (Human Plasma-Derived Coagulation Factor VIII Concentrate) in Hemophilia A Patients

Baxalta

Status and phase

Completed

Phase 3

Conditions

Hemophilia A

Treatments

Drug: Human Plasma-Derived Coagulation Factor VIII Concentrate (Virus Inactivated by Polysorbate 80 Treatment and Vapor Heat Treatment)

Study type

Interventional

Funder types

Industry

Identifiers

NCT00162019

040201

Details and patient eligibility

About

The purpose of this study is to evaluate whether IMMUNATE S/D is effective and safe in the treatment of hemophilia A patients. The study consists of 3 parts: Part 1 is a pharmacokinetic comparison of IMMUNATE S/D and its predecessor IMMUNATE. Part 2 is an evaluation of efficacy and safety of IMMUNATE S/D. Part 3 is a pharmacokinetic study of IMMUNATE S/D.

Enrollment

56 patients

Sex

Male

Ages

12 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Plasma factor VIII level as follows: for Parts 1 & 3: Subjects with severe hemophilia A (plasma baseline factor VIII level <= 1% measured at time of screening) for Part 2: Subjects with severe (plasma baseline factor VIII level <= 1% measured at time of screening) or moderately severe hemophilia A (plasma baseline factor VIII level <= 2% measured at time of screening)

Males >= 12 but <= 65 years of age
>= 35 kg body weight
Previously treated with factor VIII concentrate(s) for a minimum of 150 exposure days (as documented in the subject's medical history)
Evidence of a protective titer to HAV and HBV at the time of screening
Immunocompetent as defined by a CD4+ lymphocyte count >400/mm3 and an absolute neutrophil count (ANC) >1500
Signed informed consent obtained from subject or legally authorized representative

Exclusion criteria

Documented history of inhibitor to factor VIII with a titer >= 0.8 BU
Current evidence of inhibitor to factor VIII with a titer >= 0.8 BU, measured at the time of screening
Abnormal renal function (serum creatinine > 1.5 mg/dL)
HIV-seropositive individuals with any of the following at the time of screening:
CD4+ lymphocyte count >400/mm3
AIDS-related complex
symptomatic AIDS Note: HIV-seropositive subjects with an absolute CD4+ lymphocyte count > 400/mm3 are eligible to participate. HIV-seropositive subjects receiving highly active anti-retroviral therapy (HAART) regimens are eligible for enrollment if they are not excluded by the above criteria
Active hepatic disease (ALT and AST levels > 5 times the upper limit of normal)
Clinical or laboratory evidence of hepatic cirrhosis including (but not limited to) a recent and persistent INR (international normalized ratio) > 1.4, the presence of splenomegaly and/or significant spider angiomata on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
Known hypersensitivity to IMMUNATE
The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 30 days of study entry
The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids at a dose greater than 10 mg/day)
The subject is identified by the investigator as being unable or unwilling to perform study procedures