Severe Hemophilia A
Conditions
Interventions
This is a prospective, multicenter, multinational, non-interventional, longitudinal study in male participants major than 18 years who have been diagnosed with severe Hemophylia A treated continuously
Other
Sponsors
BioMarin Pharmaceutical Inc.
UNICAMP - Campus Campinas
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Males major of 18 years of age with hemophilia A and residual FVIII levels minor or equal 1 IU/dL, as evidenced by medical history, at the time of signing the informed consent. Must have been on prophylactic FVIII replacement therapy for at least 6 months prior to study entry. High-quality, well-documented historical data concerning bleeding episodes and FVIII usage over the previous 6 months must be available. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs). Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study-related procedures. No history of FVIII inhibitor, and results from a Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions (the most recent one of which should be tested at the central laboratory) at least one week apart previously. Known status of antibodies against the AAV5 capsid. Participants may be either AAV5- or AAV5+. HIV-positive patients may be enrolled, only if the patient has a CD4 count > 200/mm3 and an undetectable viral load. Have the ability to comply with protocol requirements in the opinion of the Investigator.
Exclusion criteria
Exclusion criteria: Significant liver dysfunction with any of the following abnormal laboratory results: ALT (alanine transaminase) or AST >2X upper limit of normal (ULN); Total bilirubin >2X ULN; Alkaline phosphatase >2X ULN; or INR (international normalized ratio) major or equal 1,4 Patients whose liver laboratory assessments fall outside of these ranges may undergo repeat testing and, if eligibility criteria are met on retest, may be enrolled after confirmation by the Medical Monitor. In addition, patients with abnormal laboratory results related to confirmed benign liver conditions (eg, Gilbert’s syndrome) are considered eligible for the study notwithstanding their abnormal laboratory results and may be enrolled after discussion with the Medical Monitor. Prior liver biopsy showing significant fibrosis of 3 or 4 as rated on a scale of 0-4 on the Batts-Ludwig (Batts 1995) or METAVIR (Bedossa 1996) scoring systems, or an equivalent grade of fibrosis if an alternative scale is used. Liver cirrhosis of any etiology as assessed by prior liver ultrasound. Chronic or active hepatitis B as evidenced by positive serology testing and confirmatory HBV DNA testing. Refer to the Centers for Disease Control (CDC) table for the interpretation of serological test results in the Laboratory Manual. Active hepatitis C, as evidenced by detectable HCV RNA or currently on antiviral therapy. Active malignancy, except non-melanoma skin cancer. History of hepatic malignancy. Evidence of any bleeding disorder not related to hemophilia A. Has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant’s ability to comply with protocol requirements, the participant’s wellbeing or safety, or the interpretability of the participant’s clinical data. Prior treatment with any vector or gene transfer agent. Concurrent enrollment in another clinical study, unless it is an observational (noninterventional) clinical study that does not interfere with the requirements of the current protocol and with prior consultation with the Medical Monitor. Unwilling to receive blood or blood products for treatment of an AE and/or a bleeding episode.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To document the number and type of bleeding episodes in severe HA patients under routine clinical practice and to estimate the number of bleeding episodes over time. | — |
Secondary
| Measure | Time frame |
|---|---|
| To document treatment of severe HA patients under routine clinical practice;To collect information on patient-reported outcomes (PROs) in severe HA patients under routine clinical practice;To collect information on safety (AEs) in severe HA patients under routine clinical practice | — |
Countries
Brazil, France, Germany, Israel, Italy, Japan, South Africa, Spain, United Kingdom, United States
Contacts
Public ContactEduardo Pizolato
PSI CRO Farma Suporte Brasil Ltda.
Outcome results
None listed