Research

BCDI is committed to providing the most cutting edge treatment opportunities and for bleeding disorder patients. To achieve this commitment, BCDI participates in a variety of clinical trials and data collection studies for bleeding disorder patients.

What is a clinical trial?
Click here to download more information about clinical trials.

What is informed consent?
Click here to download more information about informed consent.

Clinical Trails

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

Antithrombin deficiency

• ATN-106: A multicenter, prospective, open-label, uncontrolled Phase 3 study to assess the efficacy, safety and PK of Atenativ in patients with congenital antithrombin deficiency undergoing surgery or childbirth.
  https://clinicaltrials.gov/ct2/show/NCT04918173term=Atenativ&cond=Antithrombin&draw=2&rank=1

Hemophilia A

Adult Patients

• SPK-8016-101 – Dose finding study of APK-8016 gene therapy in patients with hemophilia A to support future evaluations in individuals iwth FVIII inhibitors.
  https://clinicaltrials.gov/ct2/show/NCT03734588?term=gene+therapy&cond=Hemophilia+A&draw=3&rank=18

Hemophilia A & B

Pediatric & Adult

• JSBPUS – Joint Status and Bleeding Phenotype Characterization in Severe and non-severe Hemophilia A & B. Comparison of bleeding phenotype, Bleeding assessment, Beighton score & Joint ultrasound over a 3 year period.

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
• VIP- Von Willebrand Factor In Pregnancy (VIP) Study: A Multicenter Study Of Wilate Use In Von Willebrand Disease For Childbirth.
  https://Clinicaltrials.Gov/Ct2/Show/NCT04146376?Term=Pregnancy&Cond=Von+Willebrand+Disease&Draw=2&Rank=1

Immune Thrombocytopenia Purpura (ITP)

Adult Patients

• AVA-ITP-401: Prospective, Multi-center, Open-label Study Measuring Safety and Treatment Satisfaction in Adult Subjects with Chronic Immune Thrombocytopenia (ITP) after switching to Avatrombopag from Eltrombopag or Romiplostim.
  https://clinicaltrials.gov/ct2/show/NCT04638829?term=Avatrombopag&cond=ITP&draw=2&rank=2

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

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.

The following Data Collection Studies are currently being done at BCDI. Click the links below to learn more information.

Von Willebrand

• ATHN 9 – VWD Natural History Study: Safety and effectiveness of the treatments used for people with von Willebrand Disease.
• VWD ABC- The von Willebrand disease and Bleeding correlation study.

General Bleeding/Clotting Disorders

• ATHN 10 – Rare Coagulation Disorders Project – Genetic testing for specific rare bleeding disorder patient who carry a rare diagnosis
• 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