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

November 7, 2007
February 17, 2014
September 2007
December 2016   (final data collection date for primary outcome measure)
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 ]
  • 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).
  • Assessment of the safety and tolerability of Alphanate®.
  • The primary objectives of this clinical study are to:
Complete list of historical versions of study NCT00555555 on ClinicalTrials.gov Archive Site
  • 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 ]
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.
Not Provided
Not Provided
 
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
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

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.

Interventional
Phase 4
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Von Willebrand Disease
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).
Experimental: Coagulation FVIII/VWF
Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT
Intervention: Biological: Alphanate SD/HT
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
15
March 2017
December 2016   (final data collection date for primary outcome measure)

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.
Both
7 Years and older
No
Contact: Paul J Pinciaro, PhD 410-814-7617 paul.pinciaro@grifols.com
United States
 
NCT00555555
GBI 07-03
Yes
Grifols Biologicals Inc.
Grifols Biologicals Inc.
Not Provided
Study Director: Paul J Pinciaro, PhD Grifols Biologicals Inc.
Grifols Biologicals Inc.
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP