Clinical Trials

Children’s Hospital Los Angeles

    To find out more information about these trials and to see if you qualify, please contact: coagresearch@chla.usc.edu

  • Long-acting factor replacement therapy for previously untreated patients (PUPs)
  • Subcutaneous treatment for hemophilia A/B patients with and without inhibitors
  • Understanding the impact of hemophilia on patient’s neurocognitive development
  • Gene therapy for severe or moderately severe hemophilia B patients
  • TEG technology to learn more about effects of bypassing agents with Hemlibra
  • Observing patients undergoing minor surgical procedures while on Hemlibra
  • Comparing factor levels between one step clotting and chromogenic assay
  • eTHINK is a Large Clinical Study

    eTHINK is a large clinical study looking at brain function and behavior in 500-700 children and adolescents with hemophilia ages 1-21 in the USA. The study involves a single 2-3 hour visit with a neuropsychologist. There are no medications or lab tests. Participating families will be able to see their individual results. The study is sponsored by Novo Nordisk in collaboration with an external steering committee.

    For information on this topic, please see a short VIDEO featuring a hematologist, neuropsychologist, and mom of two sons with hemophilia available at www.eTHINKawarenessvideo.com.

    If you’re interested in participating, please see a list of participating sites at https://www.clinicaltrials.gov/ct2/show/NCT03660774

Orthopaedic Hemophilia Treatment Center, Los Angeles, CA

    For more information on these or any other studies, please contact either Manny Mangilit or Dr. Quon at (213) 742-1402 or email: erikabocanegra@mednet.ucla.edu

  • The ATHNdataset, a limited data set related to Bleeding and Clotting Disorders (Open to all ages/patients with bleeding disorders)
  • CDC Public Health Surveillance for Bleeding Disorders - Registry for Bleeding Disorders Surveillance (Open to all ages/patients with bleeding disorders)
  • A Multicenter Study of Eradication of Hepatitis C Virus (HCV) Infection in People with Hemophilia and Bleeding Disorders (ATHN 6) (Open to patients with bleeding disorders with hepatitis C, recently treated or untreated looking to be treated)
  • A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia (ATHN 7) (Open to all ages/All patients with Hemophilia A & B)
  • Hemophilia B patients all ages: Musculoskeletal Ultrasound for the Joint Assessment of People with Bleeding Disorders (Open to adult patient with hemophilia B)
  • An Observational Study of Postoperative Deep Venous Thrombosis (DVT) in Hemophilics Undergoing Major Orthopedic Surgery (Open to hemophilia A and B patients undergoing surgery)
  • Biomarin: A Prospective Non-Interventional Study of Bleeding Episodes, Factor VIII Infusions, and Patient-Reported Outcomes in Individuals with Severe Hemophilia A (Open to only severe hemophilia A patients)
  • Biomarin: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions (Open to only severe hemophilia A patients)
  • Biomarin: Global Prospective Laboratory Study for Adenovirus-Associated Virus (AAV) Seroprevalence in Hemophilia A Patients with Residual Factor VIII Levels ≤2 IU/dL Who Were Previously Treated with Factor VIII Concentrates (Open to only hemophilia A patients)
  • Baxalta/Takeda: A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults with Hemophilia (Open to only hemophilia A patients)
  • Baxalta/Takeda: A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion (Open to only severe hemophilia A patients)
  • UniQure: Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B (Open to only severe adult hemophilia B patients)

Center for Inherited Blood Disorders, Orange, CA

    To find out more information about these trials and to see if you qualify, please contact: Nicole Crook at ncrook@c3dibd.org

  • ATHNdataset, a Limited Data Set Related to Bleeding and Clotting Disorders - American Thrombosis and Hemostasis Network – database for all regions across the US for all bleeding and clotting patients.- All ages
  • CDC Public Health Surveillance for Bleeding Disorders-Registry for Bleeding Disorders Surveillance – Bleeding patients that have received any type of blood products or factor products. - All ages
  • A Non-Controlled, Open Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment. Dx: Hemophilia A with first diagnosis of inhibitors – all ages
  • The Implementation, Use Testing and Validation fo the Web-based Application for the Population Pharmacokinetic Service-Hemophilia (WAPPS-HEMO) - Phase II – Dx: Factor VIII or Factor IX - all ages
  • ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX – Factor VIII or Factor IX with inhibitors – 12 years and older
  • ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX. – Factor VIII or Factor IX without inhibitors – 12 years and older
  • eThink: Evolving Treatment of Hemophilia's Impact on neurodevelopment, Intelligence and other Cognitive Functions. Non-interventional study – Factor VIII and Factor IX with or without inhibitors – Ages 1-3.6 years, and 4 to 21.11 years
  • Phase III Prospective, Randomized, Crossover Trial Comparing Von Willebrand factor (rVWF) and Transexamic Acid (TA, Lysteda) to Minimize Menorrhagia in Women with Type 1 Von Willibrand Disease (VWD)- Females with VWD- Ages 18-45 years
  • California Sickle Cell Data Collection Project – Data collection on our Sickle Cell population
  • Phase 3 of SPK 8011 A Multicenter, Observational Study in Males with Hemophilia A (goes along with their Gene therapy trial they are opening later this year) – Severe Factor VIII deficiency without inhibitors, 18 years and older