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
- Registration Number
- NCT03754790
- Lead Sponsor
- Genzyme, a Sanofi Company
- 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).
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 281
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Fitusiran Fitusiran Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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 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
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
Trial Locations
- Locations (79)
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
Scroll for more (69 remaining)Phoenix Childrens Hospital Site Number : 8400009🇺🇸Phoenix, Arizona, United States