Overview
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
- To characterize the long-term efficacy of fitusiran as assessed by the frequency of:
- Bleeding episodes
- Spontaneous bleeding episodes
- Target joint bleeding episodes
- To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants 17 years of age
Description
The estimated total time on study for each participant is up to 55 months which consists of a screening period of up to 30 days, an open label treatment period of up to 48 months and a follow up period of up to 6-month after the last dose of fitusiran
Eligibility
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:
- 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
- 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 upper limit of normal reference range (ULN) for patients who are nave to fitusiran at study start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the parent study
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.