A Phase 1, Prospective, Open Label, Two Period, Fixed Sequence, Dose-Escalation Study of the PK and Safety of BAX 826 (PSA-rFVIII) in Previously Treated Patients with Severe (FVIII <1%) Hemophilia A: Protocol number: 291501
- Conditions
- Hemophilia A10035534bleeder's disease
- Registration Number
- NL-OMON43447
- Lead Sponsor
- Baxter
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
1. Previously treated male subjects aged 18 to 65 years (inclusive) at the time of screening.
2. Prior diagnosis of severe hemophilia A (FVIII level <1%) or as confirmed by the central laboratory at screening.
3. Previously treated with FVIII concentrates for *150 documented EDs.
4. Karnofsky performance score of *60
5. Human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and cluster of differentiation 4 (CD4+) count *200 cells/mm3, as confirmed by the central laboratory at screening
6. Hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing as confirmed by the central laboratory at screening; or HCV+ with chronic stable hepatitis as assessed by the investigator
7. Able to understand and have provided written informed consent including signature on an informed consent form (ICF) approved by an ethics committee (EC)
8. Have provided written authorization for use and disclosure of protected health information
9. Agree to abide by the study schedule and to return for the required assessments
10. Willing and able to comply with the requirements of the protocol
1. Detectable FVIII inhibitor at screening, with a titer *0.6 BU (Nijmegen modification of the Bethesda assay) as determined at the central laboratory
2. Documented history of FVIII inhibitors with a titer *0.4 BU (using the Nijmegen modification of the Bethesda assay or *0.6 with Bethesda assay) at any time prior to screening
3. Known clinical hypersensitivity towards mouse or hamster proteins or to PSA
4. Scheduled elective surgery during study participation
5. Severe chronic hepatic dysfunction (defined as *5 x upper limit of normal [ULN] alanine aminotransferase [ALT] or an international normalized ratio [INR] >1.5).
6. Severe renal impairment (serum creatinine >2.0 mg/dL)
7. Currently receiving, or has recently received (less than 3 months prior to study participation), or is scheduled to receive during the course of the study, other PSA-ylated drugs
8. Have received another investigational drug within 30 days prior to study entry and/or is scheduled to receive additional investigational drug during the course of the study in the context of another investigational drug study
9. Diagnosis of an inherited or acquired hemostatic defect other than hemophilia A
10. Currently receiving, or scheduled to receive during the course of the study, an immune-modulating drug (eg, systemic corticosteroid agent at a dose equivalent to hydrocortisone greater than 10 mg/day, or alpha interferon) other than anti-retroviral chemotherapy
11. Has a clinically significant medical, psychiatric or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect the safety or compliance of the subject during the study
12. Is a family member or employee of the investigator
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method