BCDI is committed to providing the most cutting edge treatment opportunities 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 Trials

The following clinical trials are currently available at BCDI.

 

Hemophilia Studies:

  • JSBUS-BCDI XI – Joint Status and Bleeding Phenotype Characterization in Severe and Non-Severe Hemophilia A and B.
  • OBS17523 – Prospective, Observational, Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients with Hemophilia A.
  • AHAEmi – Emicizumab in Acquired Hemophilia Patients.
  • SANOFI – IQVIA: ACTIVIIITY –  Prospective, observational study of the impact of efanesoctocog alfa (ALTUVIIIO®) on goal attainment & physical activity in people with moderate or severe Hemophilia A.
  • CHESS US Study – The Cost of Hemophilia in Men: a Socio-Economic Survey US.

 

Antithrombin Studies:

  • 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 delivery.

 

ITP Studies:

  • PRN1008-010B – Principia ITP Study.
  • SCINR- Severe Neutropenia International Registry Protocol.
  • A Retrospective Chart Review of Chronic ITP Patients that Received Experimental or Off-Label Therapy for Treatment of ITP

 

Von Willebrand Disease Studies:

  • BCDI-XII – Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A patients.
  • Baxalta 071102 – Phase 3, prospective, multicenter, uncontrolled open-label clinical study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without Advate in the treatment and control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the PK of rVWF in Elective and Emergency surgeries, and the PK of rVWF in Children Diagnosed with Severe VWD.
  • Blood works VIP – Von Willebrand Factor in Pregnancy (VIP) Study: A Multicenter Study of Wilate Use in von Willebrand Disease for Childbirth.
  • Zimmerman: Molecular and Clinical Biology of VWD, study the relationship between genetic defects of VWF or related genes on the diagnosis and management of VWD.

 

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.

ATHN Transcends:

  • A natural History cohort study of the safety effectiveness, and practice of treatment in people with non-neoplastic hematologic disorders.

CDC/Registry for bleeding disorders:

  • Provides inhibitor testing for Hemophilia patients and vWD type 3.

ATHN data set: 

  • HTC patients with inherited bleeding disorders will give authorization to collect and store data in a nationwide database.  This database will collect information on bleeding disorders of select patients and the treatments they receive at the HTC.

ATHN 10:

  • Rare Coagulation Disorders Project: Free genetic testing for specific rare bleeding disorder patients who already carry a diagnosis.