Hemophilia
Conditions
Interventions
Drug: Fitusiran
Pharmaceutical form: solution for injection Route of administration: subcutaneous
Sponsors
Sanofi K.K.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Participant must be at least 12 years of age inclusive, at the time of signing the informed consent - Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial - Male - Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative
Exclusion criteria
Exclusion criteria: - Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis - Current participation in immune tolerance induction treatment (ITI) - Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period - Use of compounds other than factor concentrates or BPAs for hemophilia treatment - Current or prior participation in a gene therapy trial - Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) >1.5 x upper limit of normal reference range (ULN) for patients who are naive to fitusiran at study start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the parent study - Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort: - - Clinically significant liver disease - - History of arterial or venous thromboembolism
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Number of participants with treatment emergent adverse events (TEAEs) [Time Frame: from study baseline (day 1) up to maximum 88 months] The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Annualized bleeding rate (ABR) [Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months] Annualized bleeding rate (ABR) in the treatment period 2. Annualized spontaneous bleeding rate [Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months] Annualized spontaneous bleeding rate in the treatment period 3. Annualized joint bleeding rate [Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months] Annualized joint bleeding rate in the treatment period 4. Changes in Haem-A-quality of life (QoL) score [Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months] Change in Haem-A-QoL physical health score in the treatment period (in participants >=17 years of age) from first ever dose of fitusiran (day 1) to month 48 | — |
Countries
Australia, Canada, China, Denmark, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Malaysia, Republic of Korea, South Africa, Taiwan, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactUnit Clinical
Sanofi K.K.
Outcome results
None listed