Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

Phase 4Active Not RecruitingNCT00555555

Grifols Biologicals, LLC15 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Von Willebrand Disease

Interventions

Alphanate SD/HTBIOLOGICAL

A general guideline based on the product Full Prescribing Information is recommended with a maximum dose of 80 VWF:RCof IU/kg. The number of administrations before, during, and after the surgery procedure depends on the subject's clinical condition and the type of surgery itself. Single administrations or multiple doses may be appropriate. The dose of Alphanate® administered to each subject will be recorded as IU of VWF:RCof and also as IU of FVIII:C. The lot number for each vial of concentrate administered will also be recorded.

Eligibility

Sex: ALLMin age: 7 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Male or female 7 years of age or older 2. The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's medical records. 3. The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as "Major" according to the criteria of the protocol). 4. The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator's judgment. 5. 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: 1. The subject has been diagnosed of acquired VWD. 2. The subject is known to have history of intolerance to any Alphanate® containing substance. 3. The subject is known to have history of anaphylactic reaction(s) to blood or blood components. 4. Liver function tests (AST, ALT, bilirubin) \> 2.5 x upper limit of normal (ULN). 5. Renal function test (creatinine, BUN) \> 1.5 x ULN. 6. The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF. 7. The subject is known to abuse alcohol or illicit drug use within the past 12 months. 8. 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. 9. 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

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.

Time frame: 1 Day

Assessment of Safety and Tolerability

Time frame: 30 days

Data from ClinicalTrials.gov

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