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

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]