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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Grifols Biologicals Inc.
Sponsor:
Information provided by (Responsible Party):
Grifols Biologicals Inc.
ClinicalTrials.gov Identifier:
NCT00555555
First received: November 7, 2007
Last updated: February 17, 2014
Last verified: February 2014
  Purpose

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 Intervention Phase
Von Willebrand Disease
Biological: Alphanate SD/HT
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Grifols Biologicals Inc.:

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). [ Time Frame: 30 days ] [ Designated as safety issue: No ]

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. [ Time Frame: 1 day ] [ Designated as safety issue: No ]
  • Assessment of Safety and Tolerability [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 15
Study Start Date: September 2007
Estimated Study Completion Date: March 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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).

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.

  Eligibility

Ages Eligible for Study:   7 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00555555

Contacts
Contact: Paul J Pinciaro, PhD 410-814-7617 paul.pinciaro@grifols.com

Locations
United States, Ohio
Cincinnati Children's Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Ann Pillow    513-636-7198    anngreene.pillow@cchmc.org   
United States, Oklahoma
Oklahoma Center for Bleeding and Clotting Disorders Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact: Sarah M Hawk, P.A. C    405-271-3661 ext 1    sarah-hawk@ouhsc.edu   
Sponsors and Collaborators
Grifols Biologicals Inc.
Investigators
Study Director: Paul J Pinciaro, PhD Grifols Biologicals Inc.
  More Information

No publications provided

Responsible Party: Grifols Biologicals Inc.
ClinicalTrials.gov Identifier: NCT00555555     History of Changes
Other Study ID Numbers: GBI 07-03
Study First Received: November 7, 2007
Last Updated: February 17, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Grifols Biologicals Inc.:
Factor VIII deficiency
Von Willebrand disease
FVIII/VWF concentrate
Bleeding disorders
Blood disorders

Additional relevant MeSH terms:
Von Willebrand Diseases
Von Willebrand Disease, Type 3
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 29, 2014