NCT00555555

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.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_4

Timeline
35mo left

Started Sep 2007

Longer than P75 for phase_4

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress87%
Sep 2007Mar 2029

Study Start

First participant enrolled

September 1, 2007

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

November 7, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 8, 2007

Completed
21.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2028

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2029

Last Updated

August 25, 2023

Status Verified

August 1, 2023

Enrollment Period

21.3 years

First QC Date

November 7, 2007

Last Update Submit

August 24, 2023

Conditions

Von Willebrand Disease

Keywords

Factor VIII deficiencyVon Willebrand diseaseFVIII/VWF concentrateBleeding disordersBlood disorders

Outcome Measures

Primary Outcomes (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).

    30 days

Secondary Outcomes (2)

  • 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

Study Arms (1)

Coagulation FVIII/VWF

EXPERIMENTAL

Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT

Biological: Alphanate SD/HT

Interventions

Alphanate SD/HTBIOLOGICAL

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.

Also known as: Alphanate® Factor VIII/VWF concentrate (Human)
Coagulation FVIII/VWF

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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 unlikely to adhere to the protocol requirements of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

von Willebrand DiseasesHemophilia AHemostatic DisordersHematologic Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHemic and Lymphatic DiseasesCoagulation Protein DisordersBlood Platelet DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesVascular DiseasesCardiovascular Diseases

Study Officials

  • Paul J Pinciaro, PhD

    Grifols Biologicals, LLC

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 7, 2007

First Posted

November 8, 2007

Study Start

September 1, 2007

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

March 1, 2029

Last Updated

August 25, 2023

Record last verified: 2023-08

Data Sharing

IPD Sharing
Will not share