BCDI is committed to provide the most cutting edge treatment opportunities for Bleeding disorder patients. To that end, BCDI participates in a variety of clinical trials for bleeding disorder patients. 


What is a clinical trial?  /filesimages/What is a Clinical Trial (4).pdf

What is informed consent?   /filesimages/What is informed consent (13).pdf


The following clinical trials are currently available at BCDI. Click on the links below to learn more information.

Hemophilia A and B

Pediatric

•Baxalta 261203 - Phase 3 prospective study of the safety and efficacy of Prophylactic BAX855 in previously un-treated patients with severe hemophilia A  <6 years old

https://clinicaltrials.gov/ct2/show/NCT02615691

Adult



Von Willebrand Disease


• Zimmerman/RO1- This study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder. This study will look at a new screening blood test used to determine if a person has VWD.

https://clinicaltrials.gov/ct2/show/NCT02466789


• Shire 071102 - A study of recombinant von Willebrand factor with or without Advate to treat and control bleeding episodes, elective and emergency surgeries in children diagnosed with severe von Willebrand disease. 
https://clinicaltrials.gov/ct2/show/NCT02932618


Venous Thrombosis 

• Pfizer B0661037 - Study Of Apixaban Versus Standard Of Care In Pediatric Patients Requiring Anticoagulation For The Treatment Of A Venous Thromboembolic Event

http://clinicaltrials.gov/ct2/show/study/NCT02464969


Immune Thrombocytopenia Purpura (ITP)

Adult Patients

• PRN1008-010: Adaptive, open-label, dose-finding, phase 1/2 study investigating the safety, pharmacokinetics and clinical activity of PRN1008, an oral BTK inhibitor, in patients with relapsed immune thrombocytopenic purpura.

https://clinicaltrials.gov/ct2/show/NCT03395210


• TAK-079: Study to evaluate safety, tolerability, and efficacy of TAK-079 in participants with persistent/chronic Primary Immune thrombocytopenia. 

http://clinicaltrials.gov/ct2/show/NCT04278924

Pediatric Patients

• AVA-PED-301:  Randomized double-blind, placebo controlled, Open-label study to evaluate the efficacy and safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric subjects with Immune Thrombocytopenia. 

https://clinicaltrials.gov/ct2/show/NCT04516967?recrs=a&cond=ITP&cntry=US&draw=2&rank=10 


Data Collection Studies gather clinical data on bleeding disorder patients and the data helps physicians develop best practices for the treatment of bleeding disorder patients.

 

Current Data collection studies available at BCDI.  Click the links below to learn more information. 


Hemophilia


HUGS VIII - Costs and impact of disease in people with Hemophilia A.


von Willibrand Disease


• ATHN 9 - VWD Natural History Study: Safety and effectiveness of the treatments used for people with von Willebrand Disease.


General Bleeding/Clotting Disorders

•ATHNDATA SET: ATHN's safe and secure national patient health data resource - the ATHNdataset - has grown to include over 23,646 patients who have chosen to "opt-in" by signing a patient authorization at their Hemophilia Treatment Center (HTC).

http://athn.org


 •CDC REGISTRY- CDC Public Health Surveillance for bleeding Disorders - Registry for bleeding disorders surveillance.

http://athn.org/?q=content/public-health-surveillance


 •SCNIR: Severe Chronic neutropenia International Registry an organization dedicated to improving understanding and treatment for diseases causing severe chronic neutropenia. 

http://depts.washington.edu/registry


•Outcomes of Regional Consensus Treatment Guidelines for Management of Surgery, Invasive Procedures and Control of Hemorrhages in Patients with Bleeding Disorders; and Management of Thrombosis and Thromboprophylaxis in patients with Thrombophilia.