Hemophilia
Conditions
Interventions
Drug: Fitusiran
Pharmaceutical form: Solution for injection, Route of administration: Subcutaneous (SC)
Drug: Clotting factor concentrates (CFC) or bypassing agents (BPA)
- Coagulation factor VIII (
Sponsors
Obara Kentaro
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Diagnosis of severe congenital hemophilia A or B (FVIII <1% or FIX level <=2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence - For participants currently not on prophylaxis (clotting factor concentrates [CFC] or bypassing agents [BPA] on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) 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 in the case of participants under the age of legal consent, per local and national requirements
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: - Known co-existing bleeding disorders other than congenital hemophilia A or B - History of arterial or venous thromboembolism, not associated with an indwelling venous access. - History of intolerance to subcutaneous (SC) injection(s). - Current participation in immune tolerance induction therapy (ITI) - Prior gene therapy - Current or prior participation in a fitusiran trial - Current or prior participation in a gene therapy trial - Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer - Presence of clinically significant liver disease - AT activity <60% at Screening - Co-existing thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis - Presence of acute hepatitis, ie, hepatitis A, hepatitis E. - Presence of acute or chronic hepatitis B infection - Known to be human immunodeficiency virus (HIV) positive with CD4 count <200 cells/micro L. - Reduced renal function
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1.Annualized bleeding rate (ABR) in the fitusiran primary efficacy period [Time Frame baseline: Day 169 to Day 505 (since the first dose of fitusiran)] A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on prophylaxis standard of care (SOC) in the SOC period [Time Frame baseline: Day 169 to Day 505 (primary efficacy period) and Day -168 to Day -1 (SOC period) ] A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding. 2.Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on on-demand standard of care (SOC) in the SOC period [Time Frame baseline: Day 169 to Day 505 (primary efficacy period) and Day -168 to Day -1 (SOC period)] A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding. 3.Annualized spontaneous bleeding rate in the fitusiran primary efficacy period and in the SOC period [Time Frame baseline: Day 169 to Day 505 (primary efficacy period) and Day -168 to Day -1 (SOC period) ] A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding. A spontaneous bleeding episode is a bleeding event that occurs for no apparent or known reason, particularly into the joints, muscles, and soft tissues. 4.Annualized joint bleeding rate in the fitusiran primary efficacy period and in the SOC period [Time Frame baseline: Day 169 to Day 505 (primary efficacy period) and Day -168 to Day -1 (SOC period) ] A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding. A joint bleeding episode is characterized by an unusual sensation in the joint ("aura") in combination with 1) increasing swelling or warmth over the skin over the joint, 2) increasing pain, or 3) progressive loss of range of motion or difficulty in usin | — |
Countries
Canada, China, France, Germany, Greece, India, Italy, Japan, Mexico, Poland, Republic of Korea, Saudi Arabia, South Africa, Spain, Taiwan, Turkey, United States
Contacts
Public ContactUnit Clinical
Sanofi K.K.
Outcome results
None listed