Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00555555
Recruitment Status : Active, not recruiting
First Posted : November 8, 2007
Last Update Posted : April 10, 2019
Information provided by (Responsible Party):
Grifols Biologicals, LLC

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.

Condition or disease Intervention/treatment Phase
Von Willebrand Disease Biological: Alphanate SD/HT Phase 4

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Post-marketing Observation Study to Assess the Efficacy and Safety of the FVIII/VWF Complex (Human,) Alphanate(R), in Preventing Excessive Bleeding During Surgery in Subjects With Congenital Type 3 Von Willebrand Disease
Actual Study Start Date : September 2007
Estimated Primary Completion Date : December 2028
Estimated Study Completion Date : March 2029

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 Name: Alphanate(R) Factor VIII/VWF concentrate (Human).

Primary Outcome Measures :
  1. 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 Measures :
  1. 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 ]
  2. Assessment of Safety and Tolerability [ Time Frame: 30 days ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00555555

Sponsors and Collaborators
Grifols Biologicals, LLC
Layout table for investigator information
Study Director: Paul J Pinciaro, PhD Grifols Biologicals, LLC

Layout table for additonal information
Responsible Party: Grifols Biologicals, LLC Identifier: NCT00555555     History of Changes
Other Study ID Numbers: GBI 07-03
First Posted: November 8, 2007    Key Record Dates
Last Update Posted: April 10, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Grifols Biologicals, LLC:
Factor VIII deficiency
Von Willebrand disease
FVIII/VWF concentrate
Bleeding disorders
Blood disorders
Additional relevant MeSH terms:
Layout table for MeSH terms
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII