Endorsed Studies

FDA/NIH Approved Research Studies


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

Investigator-Initiated Observational Studies


  • 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]

 Study Recruitment


  • Shared Decision-Making for Hemophilia Gene Therapy

    Dr. Courtney Thornburg and her research team at Rady Children’s Hospital-San Diego (RCHSD) Hemophilia Treatment Center are conducting a research study about shared decision-making for hemophilia gene therapy. The study is funded by the National Hemophilia Foundation Innovative Investigator Award and BioMarin Pharmaceuticals.

    The study is designed to develop shared decision-making tools that can be used in clinical discussions with patients with hemophilia who may be eligible for gene therapy. Dr. Thornburg and her research team developed a prototype of the Clinician Decision Box tool, and they are looking for feedback from HTC healthcare providers prior to pilot testing with patients. The research involves reviewing the tool and providing feedback during a 45-60 minute interview by telephone or video conference with a member of the research team. Participants who complete the interview will receive a $100 electronic gift card to compensate for their time.

    If you would like to participate, please contact our team via e-mail at: [email protected] or via phone at: 858-966-1700 x 227376 or complete this four-question form to provide your contact information to the study team.