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
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
281
Pharmaceutical form:solution for injection Route of administration: subcutaneous
Number of participants with treatment emergent adverse events (TEAEs)
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
Time frame: from study baseline (day 1) up to maximum 88 months
Annualized bleeding rate (ABR)
Annualized bleeding rate (ABR) in the treatment period
Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
Annualized spontaneous bleeding rate
Annualized spontaneous bleeding rate in the treatment period
Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
Annualized joint bleeding rate
Annualized joint bleeding rate in the treatment period
Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
Changes in Haem-A-quality of life (QoL) score
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
Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
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Phoenix Childrens Hospital Site Number : 8400009
Phoenix, Arizona, United States
Children's Hospital Los Angeles Site Number : 8400019
Los Angeles, California, United States
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016
Orange, California, United States
Nemours Children's Clinic Site Number : 8400008
Jacksonville, Florida, United States
St Joseph's Children's Hospital of Tampa Site Number : 8400002
Tampa, Florida, United States
Rush University Medical Center -1725 W Harrison St Site Number : 8400001
Chicago, Illinois, United States
~Massachusetts General Hospital Site Number : 8400011
Boston, Massachusetts, United States
University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012
Ann Arbor, Michigan, United States
Alliance for Childhood Diseases Site Number : 8400007
Las Vegas, Nevada, United States
Childrens Hospital Medical Center of Akron Site Number : 8400006
Akron, Ohio, United States
...and 69 more locations