Hemophilia
Conditions
Brief summary
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
Detailed description
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Interventions
DRUGFitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment emergent adverse events (TEAEs) | 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 | Description |
|---|---|---|
| Annualized bleeding rate (ABR) | from first ever dose of fitusiran (day 1) up to maximum 78 months | Annualized bleeding rate (ABR) in the treatment period |
| Annualized spontaneous bleeding rate | from first ever dose of fitusiran (day 1) up to maximum 78 months | Annualized spontaneous bleeding rate in the treatment period |
| Annualized joint bleeding rate | from first ever dose of fitusiran (day 1) up to maximum 78 months | Annualized joint bleeding rate in the treatment period |
| Changes in Haem-A-quality of life (QoL) score | from first ever dose of fitusiran (day 1) up to maximum 78 months | Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from first ever dose of fitusiran (day 1) up to maximum 78 months |
Countries
Australia, Canada, China, Denmark, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Malaysia, South Africa, South Korea, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom, United States
Outcome results
None listed