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:

  • AHAEmi – Emicizumab in Acquired Hemophilia Patients. Clinical Trail NCT05345197.
    • Dr. Roberts is the principal investigator for this study.
    • Diagnosis of AHA based on reduced FVIII activity (<50%) & positive FVIII inhibitor (>0.6 BU/ml) at screening. Currently bleeding or utilizing hemostatic agents to prevent bleeds within the last 72 hours.
  • CHESS US Study: The Cost of Hemophilia in Men: a Socio-Economic Survey US
    • Dr. Roberts is the principal investigator for this study.
    • Males, age aged ≥18 years at date of clinical consultation. Diagnosed with non-acquired haemophilia A or B of any severity or an inhibitor at the time of study recruitment.
  • JSBUS-BCDI XI – Joint Status and Bleeding Phenotype Characterization in Severe and Non-Severe Hemophilia A and B
    • Investigator-Initiated Study (IIS): Dr. Roberts Principal Investigator.
    • Severe and non-severe A or B <40% factor levels and willing to undergo MSKUS. No other bleeding disorders.
  • OBS17523 – Prospective, Observational, Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients with Hemophilia A (Advarra). Clinical Trial NCT05911763.
    • Dr. Roberts is the principal investigator for this study.
    • Hemophilia A patient, who switches to Altuviiio within 30 days of study entry. Severe Hem A patients may be eligible for Joint health cohort.
  • OBS18140 (ACTIVIIITY): Prospective, observational study of the impact of efanesoctocog alfa (ALTUVIIIO®) on goal attainment & physical activity in people with moderate or severe Hemophilia A. Clincal Trial NCT06530030.
    • Dr. Roberts is the principal investigator for this study.
    • At enrollment, newly starting prophylaxis therapy with efa according to usual clinical practice, with efa initiated within 6 months after the enrollment visit. Diagnosis of moderate (endogenous FVIII activity, 1% to 5% of normal) or severe (endogenous FVIII activity <1% of normal) hemophilia A. Aged 12 to 50 years at time of enrollment, inclusive. Access to a smartphone device with Bluetooth, internet, wear ActiGraph LEAP™ device. 

 

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. Clinical Trial NCT04918173.
    • Dr. Tarantino is the principal investigator for this study.
    • 18 < 80 yrs. old with AT deficiency with AT<60%; ≥12 – 17 yrs. olds for PK portion with AT <60%

 

ITP Studies:

  • J2N-MC-JZNZ-  A Phase 1/2 Global, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults with ITP (Advarra). Clinical Trial NCT06721013.
    • Dr. Tarantino is the principal investigator for this study.
    • Due to open June 1, 2025.
    • Documented diagnosis of primary ITP. Platelet count < 100 × 109/L in the absence of other causes or disorders that may be associated with thrombocytopenia.
  • PRN1008-010B – Principia ITP Study. Clinical Trial NCT03395210.
    • Dr. Tarantinio is the principal investigator for this study.
  • SCINR- Severe Neutropenia International Registry Protocol.
    • Dr. Tarantinio is the principal investigator for this study.
  • A Retrospective Chart Review of Chronic ITP Patients that Received Experimental or Off-Label Therapy for Treatment of ITP
    • Led by Dr. Tarantino and Dr. Jessica Mistretta.

 

Von Willebrand Disease Studies:

  • BCDI-XII – Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A patients. Clinical Trial NCT05500807.
    • Investigator-Initiated Study (IIS): Dr. Roberts Principal Investigator.
    • VWD type 3, severe VWD, or combination VWD/Hemophilia A diagnosis. Bleeding phenotype necessitating prophylaxis per treating provider. Age 0 and older (infants weighing ≥3 kg) at time of signing Informed Consent Form; in the case of a proxy, a legally authorized representative must sign. VWF Antigen, activity, or collagen level < 20 U/dl ( < 50 with hemophilia), or a variant VWD confirmed by genetic testing.
  • 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.
  • TAK-577-3001 (Takeda) – Phase 3, Prospective, Open-label Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis with rVWF in Children Diagnosed with Severe VWD (Advarra). Clinical Trial NCT05582993.
    • Dr. Roberts is the principal investigator for this study.
    • < 18 yrs, Severe VWD (baseline VWF:RCo <20 iu/dl), with a history of substitution therapy with VWF concentrate required to control bleeding and a diagnosis of Type 1, Type 2 (2A, 2B, 2M, 2N) or Type 3.
  • Blood works VIP – Von Willebrand Factor in Pregnancy (VIP) Study: A Multicenter Study of Wilate Use in von Willebrand Disease for Childbirth. Clinical Trial NCT04146376.
    • Dr. Roberts is the principal investigator for this study.
    • VWD type 1, 2, or 3 defined prepartum.  Consented to study prior to gestational week 39
  • 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.
    • Dr. Roberts is the principal investigator for this study.
    • Currently only enrolling subjects who are diagnosed with vWD, type 3

 

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 10: Rare Coagulation Disorders Project-Free genetic testing for specific rare bleeding disorder patients who already carry a diagnosis.
    • Dr. Roberts is the principal investigator for this study.
  • 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.
    • Dr. Tarantino is the principal investigator for this study.
  • ATHN Transcends: A natural history cohort study of the safety effectiveness, and practice therapies used in of treatment in people with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency (Advarra).
    • Dr. Roberts is the principal investigator for this study.
  • CDC/Registry for bleeding disorders: continuation of the CDC/UDC registry – all eligible patients on the UDC should join this continuation of the registry.
    • Dr. Tarantino is the principal investigator for this study.