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

Grifols

Status and phase: Active, not recruiting; Phase 4

Conditions: Von Willebrand Disease

Treatments: Biological: Alphanate SD/HT

Study type: Interventional

Funder types: Industry

Identifiers

NCT00555555

GBI 07-03

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 estimated patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Coagulation FVIII/VWF

Experimental group

Description:

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

Treatment:

Biological: Alphanate SD/HT