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 links provided.

Studies:

  • 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 
    View Study
  • Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B 
    View Study
  • Gene Therapy, Open-label, Dose-escalation Study of SPK-9001 [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B 
    View Study
  • Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome 
    View Study
  • Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap 
    View Study

 

INVESTIGATOR INITIATED OBSERVATIONAL STUDIES

Studies:

  • An Observational Study of the Natural History of Outcomes in Hemophilics Undergoing Major Orthopedic Surgery
    14 active sites, 102 subjects enrolled
    Principal Investigator: Nigel Key, MD  [email protected]
    Study Coordinator: Robin Kellerman, RN, BSN, MSS    [email protected]

              

 

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