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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Von Willebrand Disease
• Intervention / Treatment: Biological: Alphanate SD/HT

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 Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 4

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Coagulation FVIII/VWF; Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT

Biological: Alphanate SD/HT; 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.; Other Names: Alphanate® Factor VIII/VWF concentrate (Human)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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).	30 days

Secondary Outcome Measures

Outcome Measure	Time Frame
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
Assessment of Safety and Tolerability	30 days

Collaborators and Investigators

• Sponsor: Grifols Biologicals, LLC
• Investigators: Study Director: Paul J Pinciaro, PhD, Grifols Biologicals, LLC

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2007
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): March 1, 2029

Study Registration Dates

• First Submitted: November 7, 2007
• First Submitted That Met QC Criteria: November 7, 2007
• First Posted (Estimated): November 8, 2007

Study Record Updates

• Last Update Posted (Actual): August 25, 2023
• Last Update Submitted That Met QC Criteria: August 24, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: Factor VIII deficiency; Von Willebrand disease; Blood disorders; Bleeding disorders; FVIII/VWF concentrate
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases; Coagulants; Factor VIII
• Other Study ID Numbers: GBI 07-03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO