Research/Clinical Trials

Clinical trials are research studies conducted by physicians, or other medical personnel, to evaluate safety and effectiveness of a particular intervention — typically a treatment, prevention or detection strategy for a disease. Participants receive high-quality hemotological care — and will be among the first to benefit if a new approach is proven to work. 

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Current hematology studies: 

• Biogen 998HB303 - An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
• Baxter 855 - A Phase 3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity of BAX 855 (Pegylated Full-Length Recombinant FVIII) in Previously Treated Pediatric Patients with Severe Hemophilia A
• Bayer 14372 - Multicenter, open-label, active-controlled, randomized study to evaluate the efficacy and safety of an age-and body weight-adjusted rivaroxaban regimen compared to standard of care in children with acute venous thromboembolism  
• CDC Public Health Surveillance for Bleeding Disorders – Registry for Bleeding Disorders Surveillance
• Grifols ITP - A multi-center, prospective, open-label, clinical trial to assess the safety and the efficacy of a new intravenous immune globulin (IGIV3I Grifols 10%) in patients with idiopathic (immune) thrombocytopenic purpura
• Fall Risk Study - Identifying fall risk in patients with hemophilia
• Kids-DOTT Study - Prospective Multi-Center Evaluation of the Duration of Therapy for Thrombosis in Children (the “Kids-DOTT” Trial
• Leucine DBA - A Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients with Transfusion-Dependent Diamond Blackfan Anemia
• Mast Sickle Cell Study - Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC): A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial of MST-188 (purified poloxamer 188) Injection in Subjects with Sickle Cell Disease Experiencing Vaso-Occlusive Crisis
• My Life Our Future: A Hemophilia Genotyping Initiative Data and Sample Research Repository
• Novo Nordisk - NN7008-3809 Safety and Efficacy of turoctocog alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients with Haemophilia A
• Novo Nordisk - NN7088-3860 Efficacy and Safety of NNCC 0129-0000-1003 during Surgical Procedures in patients with Hemophilia A
• Pyruvate Kinase Deficiency (PKD) Natural History Study

Not enrolling new patients:

• **Biogen 9HB01EXT B-LONG - An Open-label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B
• **Novo Nordisk - NN7088-3859, A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A
• **Novo Nordisk - NN7088-3885 A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A