ATHNreport Spring 2009 - Research
Von Willebrand Disease
The von Willebrand Disease Prophylaxis Network is actively seeking treatment centers to join 27 other European and North American centers to explore the role of prophylaxis in clinically severe von Willebrand Disease. Investigators are actively engaging treatment centers to participate in this effort and encourage investigators that might be interested to contact Thomas Abshire at Thomas.Abshire@emory.edu, Erik Berntorp at Erik.Berntorp@med.lu.se, or SDonfield@rhoworld.com for more information.
The von Willebrand Disease Prophylaxis Network (vWD PN) was formed to investigate the role of prophylaxis in clinically severe VWD that is non-responsive to other treatments through prospective and retrospective study. The VWD International Prophylaxis (VIP) study is an initiative of the vWD PN . The primary objectives of the VIP study are to:
- Study the effect of prophylaxis on bleeding frequency
- Establish optimal treatment regimens for the predominant bleeding manifestations:
- joint bleeding;
- GI bleeding;
- epistaxis; and
- menorrhagia.
The prospective component of the VIP study is a multi-center, non-randomized study in which participants will undergo an escalation of treatment from one to three doses of VWD product per week, similar to the Canadian prophylaxis trial using dose escalation in subjects with hemophilia.
The retrospective studies are being conducted in parallel with the prospective study. They are multi-center with the following objectives:
- Examine the effect of prophylaxis on bleeding frequency in patients currently on prophylaxis; and
- Characterize the natural history of GI bleeding.
The retrospective study of the effect of prophylaxis on bleeding frequency is comprised of people on a prophylactic regimen that was initiated at least 6 months prior to enrollment. Data from subjects who have a history of GI bleeding due either to proven angiodysplasia or unexplained by other factors are being collected to document the number of bleeding episodes, management and outcome.
The VIP study has been approved by ATHN and is officially recognized by the Von Willebrand Factor SSC of the ISTH. It is registered at www.ClinicalTrials.gov. Please consider becoming involved in this groundbreaking effort.
Plasminogen Deficiency Registry – Powered by ATHN
ATHN is encouraging HTC providers to register patients with plasminogen deficiency.The registry will begin to determine the approximate number and type of patients in the U.S. and Canada who present with signs and symptoms of this disorder. The survey takes approximately 10 minutes to complete via the web-based data collection tool. No individual patient identifying information is required. Click here to go directly to the survey www.PlasminogenRegistry.org.
The data collected will serve as a foundation for developing a network of health care providers interested in information sharing and ultimately for identifying potential subjects for future clinical trials for a plasminogen replacement product. Please participate in the survey today.
Severe Factor X Deficiency Study – Centers Needed
Additional study sites are needed for an international trial of high-purity plasma-derived factor X concentrate in patients with severe factor X deficiency to determine if it will offer advantages over the current standard treatment of Prothrombin Complex concentrates (PCCs) or fresh-frozen plasma (FFP). The trial, sponsored by UK-based Bio Products Laboratory (BPL), will investigate the pharmacokinetics, safety and efficacy of a high purity factor X concentrate in patients with severe factor X deficiency. Patients that meet the study criteria are needed.
Factor X deficiency is a rare coagulation disorder and one of the most severe of the inherited coagulation disorders. Currently, there are no factor X concentrates available and FFP is normally used as treatment. Prothrombin Complex concentrates have been used in patients, but the amount of factor X in each product has not been consistent. There has also been a reported risk of thromboembolic complications with PCC product usage. BPL hopes to change treatment options with development of a high-purity plasma-derived factor X concentrate. The company has an international clinical trial underway.
Based on a feasibility study completed earlier this year by ATHN, core US clinical trial sites have been identified to participate with centers in several countries across Europe. BPL is seeking more sites to join the trial.
BPL welcomes inquiries about the trial from clinicians treating one or more adult or pediatric patients with severe or moderate hereditary factor X deficiency. Please contact ATHN at info@athn.org or Miranda Norton, BPL Clinical Study Manager, at miranda.norton@bpl.co.uk or phone on +44 20 8258 2661 for more information.
