A Study With OPTIVATE® in People With Von Willebrand Disease

NCT00387192 | Terminated Phase 3

• 1 other identifier: 8VWF01
• Study Type: interventional
• Target: 26
• Locations: 2 countries
• Sites: 4

Brief Summary

The main objective of the study is to assess the pharmacokinetics of OPTIVATE® after a single dose of 80 IU/kg VWF:RCo. The secondary objectives of the study are to assess efficacy and safety of OPTIVATE® in long-term use over at least 12 months.

Conditions

Von Willebrand Disease

Keywords

Von Willebrand Disease; Factor VIII; Von Willebrand Factor; Pharmacokinetics

Outcome Measures

Primary Outcomes (1)

• Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.

  Baseline vist

Interventions

Optivate DRUG

Plasma-derived Factor VIII

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Have given written informed consent.
• Be aged 12 years or older.
• Have severe VWD (VWF:RCo \<20%) of known type. Severity will be confirmed by a current VWF:RCo result of \<20%.
• Be known or expected to require a concentrate for management of VWD.
• Must have had at least one bleed in the last 12 months which required treatment with a FVIII and VWF concentrate.
• Have a known lack of, or poor response to, DDAVP.
• Have a prothrombin time (PT) of not more than 3 seconds above the upper limit of the reference range.
• At the Baseline Visit (Visit 1), patients must have had at least 5 days since their last infusion of replacement factor concentrate or DDAVP.
• Female patients of child-bearing potential must have a negative result on a human chorionic gonadotropin-based pregnancy test. If a female patient is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. Female patients must not be lactating.

You may not qualify if:

• Have a history of inhibitor development to VWF or FVIII or a positive result at screening.
• Actively bleeding (Note: the patient can enter the study once the bleed is controlled).
• Presence of major systemic illnesses: renal disease, liver disease, or neurological or psychiatric disease which would compromise the outcome of the study in the opinion of the investigator.
• Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients.
• Have a recent history of alcohol or drug abuse.
• Administration of a new chemical entity within the 4 months preceding enrolment.
• Participation in any other clinical study in which investigational or marketed drugs were employed in the 30 days preceding enrolment into this study, with the exception of the BPL clinical study Protocol 8VWF03.
• In the opinion of the investigator, the patient is unlikely to comply with the study protocol.

Sponsors & Collaborators

• Bio Products Laboratory: lead

Study Sites (4)

Rambam Health Care Campus, 8 Haaliya St., Bat-Galim

Haifa, 31096, Israel

Location

Haddasah Ein-Karem Medical Center, P.O.Box 12000

Jerusalem, 91120, Israel

Location

Beilinson Hospital, Rabin Medical Center, 39 Jabontinsky Street

Petah Tikva, 49100, Israel

Location

University Department of Haematology

Manchester, M13 9WL, United Kingdom

Location

Related Links

• Sponsor's homepage

MeSH Terms

Conditions

von Willebrand Diseases; Hemophilia A

Interventions

optivate

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Charles Hay, MD

  Manchester Haemophilia Comprehensive Care Centre

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 11, 2006
• First Posted: October 12, 2006
• Study Start: November 1, 2006
• Study Completion: September 1, 2008
• Last Updated: March 3, 2010

Record last verified: 2010-03

Locations

IL (3); GB (1)