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Sponsor: Genzyme, a Sanofi Company
Conditions: Hemophilia
Interventions: Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
Countries: United States, Australia, China, Denmark, France, Ireland, Israel, Italy
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA) prophylaxis. Secondary Objectives: * To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis: * the frequency of spontaneous bleeding episodes * the frequency of joint bleeding episodes * health related quality of life (HRQOL) in participants greater than or equal to (\>=) 17 years of age * To characterize the frequency of bleeding episodes during the onset and treatment periods in participants receiving fitusiran. * To characterize the safety and tolerability of fitusiran. * To characterize the annualized weight-adjusted consumption of factor/BPA while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis.
Sex: MALE
Age: 12 Years to —
Healthy volunteers: No
Study type: INTERVENTIONAL
Inclusion Criteria: * Males, \>=12 years of age. * Severe hemophilia A or B (as evidenced by a central laboratory measurement at screening or documented medical record evidence of FVIII less than (\<) 1 percent (%) or FIX level less than or equal to (\<=) 2%). * A minimum of 2 bleeding episodes required BPA treatment within the last 6 months prior to screening for participants with inhibitory antibodies to factor VIII or factor IX (Cohort A). A minimum of 1 bleeding episode required factor treatment within the last 12 months prior to screening for participants without inhibitory antibodies to factor VIII or factor IX (Cohort B). * Met either the definition of inhibitor or non-inhibitor participant as below: * Inhibitor: Use of BPAs for prophylaxis and for any bleeding episodes for at least the last 6 months prior to screening, and met one of the following Nijmegen-modified Bethesda assay results criteria: * Inhibitor titer of \>=0.6 Bethesda Unit per milliliter (BU/mL) at screening, or * Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 2 consecutive titers \>=0.6 BU/mL, or * Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of anamnestic response * The subgroup of participants in Cohort A participants might additionally meet the following criteria to be eligible to start treatment with fitusiran directly after the screening period: * Hemophilia B with inhibitory antibody to Factor IX as defined above * Not responding adequately to BPA treatment (historical ABR \>=20) prior to enrollment * In the opinion of the Investigator, with approval of Sponsor Medical Monitor, 6-month BPA prophylaxis period should be omitted. * Non-inhibitor: Use of factor concentrates for prophylaxis and for any bleeding episodes for at least the last 6 months prior to screening, and met each of the following criterion: * Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening and * No use of BPAs to treat bleeding episodes for at least the last 6 months prior to screening and * No history of immune tolerance induction therapy within the past 3 years prior to screening. * Documented prophylactic treatment with factor concentrates or BPAs for the treatment of hemophilia A or B for at least 6 months prior to screening. * Adherent to the prescribed prophylactic therapy for at least 6 months prior to screening per Investigator assessment. * Willed and complied with the study requirements and to provide written informed consent and assent. Exclusion Criteria: * Known co-existing bleeding disorders other than hemophilia A or B. * AT activity \<60% at screening. * Co-existing thrombophilic disorder. * Clinically significant liver disease. * Active Hepatitis C virus infection. * Acute or chronic Hepatitis B virus infection. * HIV positive with a CD4 count of \<200 cells per microliter. * History of arterial or venous thromboembolism. * Inadequate renal function. * History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc). * History of intolerance to subcutaneous injection(s). * Any other conditions or comorbidities that made the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.
- Los Angeles, California, United States
- Prahran, Australia
- Beijing, China
- Copenhagen, Denmark
- Lyon, France
- Crumlin, Ireland
- Ramat Gan, Israel
- Milan, Italy
- Nagoya, Japan
- Nishinomiya, Japan
- Saitama, Japan
- Tokyo, Japan
- Ampang, Malaysia
- Johor Bahru, Malaysia
- Kota Kinabalu, Malaysia
- San Pablo, Mexico
- Busan, South Korea
- Daejeon, South Korea
- Seoul, South Korea
- Adana, Turkey (Türkiye)
- Ankara, Turkey (Türkiye)
- Antalya, Turkey (Türkiye)
- Gaziantep, Turkey (Türkiye)
- Istanbul, Turkey (Türkiye)
- Izmir, Turkey (Türkiye)
- Izmir, Turkey (Türkiye)
- Kayseri, Turkey (Türkiye)
- Samsun, Turkey (Türkiye)
- Van, Turkey (Türkiye)
- Kyiv, Ukraine
- + 5 more on CT.gov