Endorsed Studies


Information on the following studies can be found at www.clinicaltrials.gov. Search by title or click on links provided.


  • Phase 3b, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients from Birth to 12 Months of Age with Hemophilia A Without Inhibitors (HAVEN 7)
  • Phase 3 Randomized, Open-Label, Active-Controlled Study to Evaluate the Efficacy and Safety of Crovalimab Compared with Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are Currently Treated with Complement Inhibitor (COMMODORE 1)
  • Phase IV, Multicenter, Open-Label Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants with Severe or Moderate Hemophilia A without Factor VIII Inhibitors on Emicizumab Prophylaxis (Beyond ABR)
  • An Open Label Dose-escalation Study of a Self-complementary Adeno-associated Viral Vector (scAAV2/8-LP1-hFIXco) for Gene Transfer in Subjects with Hemophilia B 
  • Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B 
  • Gene Therapy, Open-label, Dose-escalation Study of SPK-9001 [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B 
  • Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome 
  • Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap 




  • An Observational Study of the Natural History of Outcomes in Hemophilics Undergoing Major Orthopedic Surgery
    13 active sites, 119 subjects enrolled
    Principal Investigator: Nigel Key, MD  [email protected]
    Study Coordinators:
    Marcus Layer, BA  [email protected] and
     Robin Kellerman, RN, BSN, MSS    [email protected]