A Post-marketing Observational Study to Assess the Efficacy and Safety of the FVIII/VWF Complex (Human), Alphanate®, in Preventing Excessive Bleeding During Surgery in Subjects With Congenital Type 3 Von Willebrand Disease
Trial Snapshot
- Phase
- Phase 4
- Status
- Active, not recruiting
- Sponsor
- Grifols Biologicals, LLC
- Enrollment
- 15
- Primary Endpoint
- Assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures (mostly major surgeries).
Study Overview
Brief Summary
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
Detailed Description
For the treatment of surgical procedures the intended dose of Alphanate® will be given as a single dose or as multiple doses over several days, depending on the clinical situation, and according to the Full Prescribing Information guideline and the investigator's judgment. For each treated event, the subject's treatment period will be finished when, in the opinion of the local Investigator, the participating subject would not benefit from further infusions of the study concentrate.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 7 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male or female 7 years of age or older
- •The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's medical records.
- •The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as "Major" according to the criteria of the protocol).
- •The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator's judgment.
- •The subject freely gives written informed consent. Patients who are not legally permitted to provide written consent must sign a form of assent for study participation, and written consent must be provided by a parent or legal guardian.
Exclusion Criteria
- •The subject has been diagnosed of acquired VWD.
- •The subject is known to have history of intolerance to any Alphanate® containing substance.
- •The subject is known to have history of anaphylactic reaction(s) to blood or blood components.
- •Liver function tests (AST, ALT, bilirubin) > 2.5 x upper limit of normal (ULN).
- •Renal function test (creatinine, BUN) > 1.5 x ULN.
- •The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF.
- •The subject is known to abuse alcohol or illicit drug use within the past 12 months.
- •The subject is participating in another clinical study involving an investigational treatment, or participated within the past 4 weeks (except if the patient is participating in another Alphanate® study). Studies consisting of data and blood sampling collections on a regular or long-term basis are exempt from this exclusion.
- •The subject is unlikely to adhere to the protocol requirements of the study.
Outcomes
Primary Outcomes
Assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures (mostly major surgeries).
Time Frame: 30 days
Secondary Outcomes
- Assessment of Safety and Tolerability(30 days)
- To assess the Day 0 (surgery day) and Day 1 (post-surgery day) treatment outcomes of each surgical procedure, rated by the investigator using a 2-point verbal rating scale.(1 Day)