Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Study on Long-term Safety and Effects of Investigational Medication in Hemophilia A and B
Study Overview
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
- To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:
- Bleeding episodes
- Spontaneous bleeding episodes
- Joint bleeding episodes
- To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Study Details
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Hemophilia
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Age: 12 years or above
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Gender: Male
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 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 × upper limit of normal reference range (ULN) for patients who are naïve 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
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
This study investigates the long-term safety and effects of an investigational medication in people with severe hemophilia A or B. Hemophilia is a condition where blood doesn't clot normally due to the lack of certain proteins. The purpose is to understand how safe the medication is over time and how it affects bleeding episodes, including spontaneous and joint-related ones.
Participants will undergo various procedures to assess the medication's impact on their health-related quality of life. These procedures include monitoring for bleeding episodes and evaluating overall well-being. The study will also gather information on any side effects experienced by the participants.
- Who can participate: Participants must be male, at least 12 years old, and have severe hemophilia A or B. They should have completed a Phase 3 trial with the investigational medication.
- Study details: Participants will receive the investigational medication and be monitored for safety and effects on bleeding episodes.
- Study timelines: The study will last 90 months.