Research HTC
Current Clinical Research – UNC Blood Research Center *as of February 13, 2024*
Active Studies – Start-Up Phase
BMN 270-401– A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
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CSL222_3003 – Long-Term Follow-Up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
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SPK-8011-302 – Phase 3 gene therapy study of SPK-8011 in adults with severe or moderately severe hemophilia A (KEYSTONESM 1)
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AHAEMI – Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-Arm, Open-Label Clinical Trial
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Active Studies – Currently Open to Enrollment
STOP-HHT – A Phase II/III randomized, placebo controlled, double blind study to evaluate the effects of up to 24 weeks of low dose pazopanib on hereditary hemorrhagic telangiectasia related epistaxis and anemia
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P200 – United States Thrombotic Microangiopathy (USTMA) Thrombotic Thrombocytopenic Purpura (TTP) Clinical Data and Biologic Sample Repository
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BVMC3 – Brain Vascular Malformation Consortium (BVMC3) Project 3: Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia
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Active Studies – Currently Following Subjects or Data Analysis Only
PATH-HHT – Placebo-controlled, double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT).
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GENEr8-1 (BMN 270-301) – Phase III, gene therapy trial for adults with severe hemophilia A.
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HOPE-B (CSL222) – Phase III, gene therapy trial for adults with severe or moderately severe hemophilia B.
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AVA-ITP-401– Phase IV trial of safety and satisfaction in adults with chronic Immune Thrombocytopenia (ITP) after switching to avatrombopag from eltrombopag or romiplostim.
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Recently Closed Studies (Jan 1 2023 – Present)
ACCRU-SC-1601 (EVE) – Randomized, phase III trial of apixaban in preventing secondary cancer related venous thrombosis.
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MOSH Outcomes – An observational study of the natural history of outcomes in hemophiliacs undergoing major orthopedic surgery.
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