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Hemophilia clinical trials at UC Davis
7 research studies open to eligible people

  • A Dose Confirmation Study of Experimental AAV5-hFIXco-Padua for Adults with Moderate-to-Severe Hemophilia B

    open to eligible males ages 18 years and up

    This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 1013 gc/kg.

    Sacramento, California and other locations

  • A Gene Transfer Study Using Experimental SPK-8011 for Hemophilia A (poor blood clotting)

    “This research study will help us learn if the study product (SPK-8011) is safe and effective in reducing bleeding for treating hemophilia A.”

    open to eligible males ages 18 years and up

    This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

    Sacramento, California and other locations

  • A Study of Experimental Human Factor 8 Gene Therapy SB-525 For Severe Hemophilia A

    open to eligible males ages 18 years and up

    The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity levels with adaptive doses of SB-525.

    Sacramento, California and other locations

  • A Study of the Long-Term Safety and Efficiency of Experimental Medicine SPK-9001 For Hemophilia B

    open to eligible males ages 18 years and up

    Long-term safety follow-up for subjects with Hemophilia B and previously treated in the C0371005 (formerly SPK-9001-101) protocol

    Sacramento, California and other locations

  • BAX 855 Previously Untreated Patient (PUP)

    open to eligible people ages up to 5 years

    The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

    Sacramento, California and other locations

  • Gene Therapy Study in Severe Haemophilia A Patients

    “Experimental treatment to correct a genetic mutation”

    open to eligible males ages 18 years and up

    This clinical trial is being conducted to learn more about a potential treatment (valoctocogene roxaparvovec) for people with severe hemophilia A. This research study will test and confirm the safety and effectiveness of the 6E13 vg/kg dose of the study drug (valoctocogene roxaparvovec) that contains the correct gene to make Factor VIII so that the body can make its own Factor VIII that functions properly. Only one dose of valoctocogene roxaparvovec is being given in this study, and this dose has been previously studied in another clinical trial in patients with hemophilia A. This is a phase 3 study which is meant to show that the study drug is safe and works to help treat hemophilia A. The study will see if liver cells are able to make Factor VIII that functions properly after receiving this study drug. The study will also examine the effects that the study drug has on how much Factor VIII concentrates patients have to inject into their veins and on their bleeding episodes after the study drug has been administered. Finally, the study will see if and how the body responds to the study drug - for example, whether liver cells become inflamed or whether the body makes antibodies (something the immune system makes to protect itself against things like bacteria and viruses) against the vector or the new Factor VIII gene.

    Sacramento, California and other locations

  • The INITIATE Research Study for people with Hemophilia A and inhibitors to Factor VIII

    “Help us learn about a patient-specific lab test to decrease time to success of Immune Tolerance Induction”

    open to eligible males

    The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex concentrate for immune tolerance induction (ITI) in subjects with congenital Hemophilia A, FVIII activity ≤2%, and a historical high-titer inhibitor [≥5 Bethesda Unit (BU)].

    Sacramento, California and other locations

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