Back to resultsEfficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
Primary Purpose
Von Willebrand Disease
Status
Active
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Alphanate SD/HT
Sponsored by
About this trial
This is an interventional treatment trial for Von Willebrand Disease focused on measuring Factor VIII deficiency, Von Willebrand disease, FVIII/VWF concentrate, Bleeding disorders, Blood disorders
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Coagulation FVIII/VWF
Arm Description
Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
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.
Assessment of Safety and Tolerability
Full Information
NCT ID
NCT00555555
First Posted
November 7, 2007
Last Updated
August 24, 2023
Sponsor
Grifols Biologicals, LLC
1. Study Identification
Unique Protocol Identification Number
NCT00555555
Brief Title
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 2007 (Actual)
Primary Completion Date
December 2028 (Anticipated)
Study Completion Date
March 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Grifols Biologicals, LLC
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand Disease
Keywords
Factor VIII deficiency, Von Willebrand disease, FVIII/VWF concentrate, Bleeding disorders, Blood disorders
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Coagulation FVIII/VWF
Arm Type
Experimental
Arm Description
Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT
Intervention Type
Biological
Intervention Name(s)
Alphanate SD/HT
Other Intervention Name(s)
Alphanate® Factor VIII/VWF concentrate (Human)
Intervention Description
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.
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
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
Title
Assessment of Safety and Tolerability
Time Frame
30 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul J Pinciaro, PhD
Organizational Affiliation
Grifols Biologicals, LLC
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
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