Hemophilia A, Hemophilia B
Conditions
Interventions
investigational material(s)
Generic name etc : SAR439774
INN of investigational material : fitusiran
Therapeutic category code : 634 Human blood preparations
Dosage and Administration for Investigatio
Sponsors
Sanofi K.K.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Inclusion Criteria: 1. Males, >=12 years of age 2. Severe hemophilia A or B without inhibitors - Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is <=2% at Screening; - On-demand use of factor concentrate to manage 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 * No use of BPAs to treat bleeding episodes for at least the last 6 months prior to Screening * No history of immune tolerance induction therapy within the last 3 years prior to Screening 3. A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening. 4. 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, ie, Von Willebrand's disease, additional factor deficiencies, or platelet disorders. - 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/microL - 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 |
|---|---|
| efficacy 1. Annualized bleeding rate (ABR) [Time Frame: 8 months (efficacy period: day 29 to Month 9)] | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy 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)] efficacy 2. spontaneous bleeding rate [Time Frame: 8 months (efficacy period: day 29 to Month 9)] efficacy 3. Annualized joint bleeding rate [Time Frame: 8 months (efficacy period: day 29 to Month 9)] efficacy 4. Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 0-100 with higher scores representing greater impairment. [Time Frame: 9 months (treatment period: 1 month onset period + efficacy period of 8 months)] efficacy 5. Number of participants reported with treatment-emergent adverse events [Time Frame: 15 months (9 months treatment + 6 months follow up)] | — |
Countries
Africa, Asia except Japan, Europe, Japan, Middle East, North America, Oceania
Outcome results
None listed