Hemophilia A, Hemophilia B
Conditions
Interventions
Drug: fitusiran
solution for injection
by subcutaneous (SC) injection
Drug: Bypassing agents
solution for injection
by intravenous (IV) injection
Sponsors
Sanofi K.K.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Inclusion Criteria: - Males, >=12 years of age - Severe hemophilia A or B with inhibitors (Severity confirmed by a central laboratory where FVIII level is =0.6 BU/mL or as evidenced by medical records) - A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening - Willing and able to comply with the study requirements and to provide written informed consent and assent
Exclusion criteria
Exclusion criteria: Exclusion Criteria: - Known co-existing bleeding disorders other than hemophilia A or B - Antithrombin (AT) activity <60% at Screening - Co-existing thrombophilic disorder - Clinically significant liver disease - Active HCV infection - HIV positive with a CD4 count of <200 cells/microlitter - 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 judgement
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1.Annualized bleeding rate(ABR) [Time Frame: 8 months (efficacy period: day 29 to Month 9)] | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Annualized bleeding rate (ABR) [Time Frame: At 1 month (onset period), 9 months (treatment period: 1 month onset period + efficacy period of 8 months)] 2.Annualized spontaneous bleeding rate [Time Frame: 8 months (efficacy period: day 29 to Month 9)] 3.Annualized joint bleeding rate [Time Frame: 8 months (efficacy period: day 29 to Month 9)] 4.Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 1-100 with higher scores representing greater impairment. [Time Frame: 9 months (treatment period: 1 month onset period + efficacy period of 8 months)] 5. Number of participants reported with treatment-emergent adverse events [Time Frame: 15 months (9 months treatment + 6 months follow up)] | — |
Countries
Australia, Canada, China, France, Germany, India, Italy, Japan, Malaysia, Republic of Korea, South Africa, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactClinical Study Unit
Sanofi K.K.
Outcome results
None listed