Hemophilia
Conditions
Interventions
Drug: Fitusiran
Pharmaceutical form: solution for injection Route of administration: subcutaneous
Drug: BPA prophylaxis
Pharmaceutical form: solution for injection Route of administration: Intravenous
Sponsors
Sanofi K.K.
Eligibility
Sex/Gender
Male
Inclusion criteria
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 Use of BPAs for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria: - Inhibitor titer of >= 0.6 BU/mL at Screening, or - Inhibitor titer of = 0.6 BU/mL, or - Inhibitor titer of = 20) prior to enrollment - - In the opinion of the Investigator, with approval of Sponsor Medical Monitor, 6-month BPA prophylaxis period should be omitted. Use of factor concentrates for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion: - Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and - No use of bypassing agents 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 bypassing agents 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 - Willing and able to comply with the study requirements and to provide written informed consent and assent
Exclusion criteria
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/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 SC injection(s) - Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Annualized bleeding rate (ABR) Annualized Bleeding Rate (ABR) in the fitusiran efficacy period and the factor or BPA prophylaxis period | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Annualized spontaneous bleeding rate Annualized spontaneous bleeding rate in the fitusiran efficacy period and the factor or BPA prophylaxis period 2. Annualized joint bleeding rate Annualized joint bleeding rate in the fitusiran efficacy period and the factor or BPA prophylaxis period 3. Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 0-100 with higher scores representing greater impairment Change in Haem-A-QOL physical health score and total score in the fitusiran treatment period 4. ABR in the onset period ABR in the fitusiran onset period 5. ABR in the treatment period ABR in the fitusiran treatment period 6. Annualized weight-adjusted consumption of factor/BPA 7. Number of patients reported with treatment emergent adverse events | — |
Countries
Australia, China, Denmark, France, Ireland, Israel, Italy, Japan, Malaysia, Mexico, Republic of Korea, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactClinical Study Unit
Sanofi K.K.
Outcome results
None listed