Hemophilia A
Conditions
Keywords
Factor VIII Deficiency
Brief summary
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation of efficacy and safety. The study is open to patients who completed Baxter Study 069901.
Interventions
Sponsors
Baxalta now part of Shire
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
10 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject has completed Baxter protocol 069901 * Subject is HIV-1 seronegative or, if HIV-1 seropositive, has a CD4+ lymphocyte count \>= 400/mm3 documented within three months of the screening visit * Subject (and his legally acceptable representative, in the case of study participants \>= 10 and \< 18 years of age) has been informed of the nature of the study, agreed to its provisions, and signed and dated the informed consent form approved by the appropriate IRB/IEC and Baxter
Exclusion criteria
* The subject received factor VIII products other than rAHF-PFM upon completion of Baxter protocol 069901 * The subject developed an inhibitor to factor VIII, measured in the central laboratory, during Baxter protocol 069901. An inhibitor is defined as a Bethesda titer \> 1.0 or, if Bethesda titer \< 1.0, confirmation using the Nijmegen modification of the Bethesda assay with a titer \> 0.6 * The subject is scheduled to receive an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids) during the course of the study * The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM) | Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion. |
Countries
Austria, Belgium, Canada, France, Germany, Italy, Sweden, United Kingdom, United States
Outcome results
None listed