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Hemophilia clinical trials at UC Health
19 studies in progress, 11 open to new patients

  • 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.

    at UC Davis

  • A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)

    open to eligible males ages 18-65

    The primary purpose is to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.

    at UCLA

  • 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.

    at UCSF UC Davis

  • A Study of Experimental Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B

    open to eligible males ages 18-65

    The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.

    at UC Davis

  • 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 SPK-9001-101 protocol

    at UC Davis

  • Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

    open to eligible males ages 18 years and up

    The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX. SB-FIX is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the Factor 9 gene into the albumin locus in hepatocytes with the goal of lifelong therapeutic production of the Factor IX clotting factor.

    at UCLA

  • Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry: A Nested Study Within an Existing Registry

    open to eligible people ages 2 years and up

    This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

    at UCSD

  • ATHN 2: Factor Switching Study

    open to all eligible people

    This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first. The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years. In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Eloctate will be approached to participate in the 'Eloctate Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.

    at UCSD

  • Dose Confirmation Trial of AAV5-hFIXco-Padua

    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.

    at UC Davis UCSD

  • Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients

    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.

    at UCSF UC Davis UCSD

  • 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)].

    at UC Davis

  • A Gene Therapy Study for Hemophilia B

    Sorry, in progress, not accepting new patients

    A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

    at UC Davis

  • A Study of Experimental Medicine "Human-cl-rhFVIII" (An Essential Blood-Clotting Protein) in Hemophilia A

    Sorry, in progress, not accepting new patients

    Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.

    at UC Davis

  • A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A

    Sorry, in progress, not accepting new patients

    This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks in participants with hemophilia A with or without inhibitors against factor VIII (FVIII). The study consists of 2 parts: a pharmacokinetic (PK) run-in part followed by an expansion part.

    at UC Davis

  • An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Ma...

    Sorry, in progress, not accepting new patients

    The primary objective of the study is to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants with severe hemophilia A. The secondary objectives are to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in previously untreated patients (PUPs), to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.

    at UCSF

  • Joint Health Study

    Sorry, not currently recruiting here

    This is a prospective, non-randomized, controlled study to examine whether or not having a higher trough during prophylactic treatment with clotting factor offers better joint protection than the standard trough of 1% Factor IX (FIX or Factor 9). This study will test the hypothesis that an extended half-life (EHL) FIX product with an intended trough of >10% could offer better protection than previous treatment concentrates. This study also examines whether or not joint damage could be diagnosed earlier using ultrasound images.

    at UCLA UCSD

  • Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.

    at UC Davis UCSD

  • Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

    Sorry, not currently recruiting here

    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 4E13 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.

    at UCSF UC Davis UCSD

  • Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

    Sorry, in progress, not accepting new patients

    The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

    at UC Davis