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)
Children's Hospital Los Angeles Site Number : 8400019
🇺🇸Los Angeles, California, 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
Childrens Hospital Medical Center of Akron Site Number : 8400006
🇺🇸Akron, Ohio, United States
Investigational Site Number : 0360001
🇦🇺Camperdown, New South Wales, Australia
Investigational Site Number : 0360002
🇦🇺Murdoch, Western Australia, Australia
Nemours Children's Clinic Site Number : 8400008
🇺🇸Jacksonville, Florida, United States
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016
🇺🇸Orange, California, United States
St Joseph's Children's Hospital of Tampa Site Number : 8400002
🇺🇸Tampa, Florida, United States
University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012
🇺🇸Ann Arbor, Michigan, United States
Investigational Site Number : 1240001
🇨🇦Montreal, Quebec, Canada
Investigational Site Number : 1560009
🇨🇳Chengdu, China
Investigational Site Number : 1560005
🇨🇳Hangzhou, China
Investigational Site Number : 1560010
🇨🇳Qingdao, China
Investigational Site Number : 3560004
🇮🇳Ranipet, India
Investigational Site Number : 3720002
🇮🇪Dublin, Ireland
Investigational Site Number : 2500002
🇫🇷Lyon, France
Investigational Site Number : 2500003
🇫🇷Paris, France
Investigational Site Number : 2760001
🇩🇪Berlin, Germany
Investigational Site Number : 2760003
🇩🇪Leipzig, Germany
Investigational Site Number : 3480002
🇭🇺Budapest, Hungary
Investigational Site Number : 3560001
🇮🇳Bangalore, India
Investigational Site Number : 3560005
🇮🇳Lucknow, India
Investigational Site Number : 3560002
🇮🇳Pune, India
Investigational Site Number : 3560010
🇮🇳Pune, India
Investigational Site Number : 3760001
🇮🇱Ramat Gan, Israel
Investigational Site Number : 3800002
🇮🇹Milano, Lombardia, Italy
Investigational Site Number : 3800004
🇮🇹Padua, Veneto, Italy
Investigational Site Number : 3920003
🇯🇵Kitakyushu, Japan
Investigational Site Number : 3920001
🇯🇵Nagoya, Japan
Investigational Site Number : 1580004
🇨🇳Taichung, Taiwan
Investigational Site Number : 4100003
🇰🇷Seoul, Seoul-teukbyeolsi, Korea, Republic of
Investigational Site Number : 4100001
🇰🇷Busan, Busan-gwangyeoksi, Korea, Republic of
Investigational Site Number : 4100002
🇰🇷Daejeon, Daejeon-gwangyeoksi, Korea, Republic of
Investigational Site Number : 4100004
🇰🇷Seoul, Korea, Republic of
Investigational Site Number : 4580003
🇲🇾Ampang, Malaysia
Investigational Site Number : 1580002
🇨🇳Changhua, Taiwan
Investigational Site Number : 7100002
🇿🇦Port Elizabeth, South Africa
Investigational Site Number : 1580008
🇨🇳Taichung, Taiwan
Investigational Site Number : 1580007
🇨🇳Taoyuan City, Taiwan
Investigational Site Number : 7920012
🇹🇷Bornova, Turkey
Investigational Site Number : 7920002
🇹🇷Adana, Turkey
Investigational Site Number : 7920004
🇹🇷Akdeniz, Turkey
Investigational Site Number : 7920006
🇹🇷Gaziantep, Turkey
Investigational Site Number : 7920007
🇹🇷Samsun, Turkey
Investigational Site Number : 7920003
🇹🇷Izmir, Turkey
Investigational Site Number : 8040001
🇺🇦Kyiv, Ukraine
Investigational Site Number : 7920008
🇹🇷Kayseri, Turkey
Investigational Site Number : 8040004
🇺🇦Mykolaiv, Ukraine
Investigational Site Number : 8040002
🇺🇦Lviv, Ukraine
Investigational Site Number : 8260004
🇬🇧Glasgow, United Kingdom
Investigational Site Number : 8260001
🇬🇧London, London, City Of, United Kingdom
Investigational Site Number : 1560004
🇨🇳Beijing, China
Investigational Site Number : 1560014
🇨🇳Changsha, China
Investigational Site Number : 1560002
🇨🇳Guangzhou, China
Investigational Site Number : 1560007
🇨🇳Beijing, China
Investigational Site Number : 1560011
🇨🇳Guiyang, China
Investigational Site Number : 1560008
🇨🇳Jinan, China
Investigational Site Number : 1560012
🇨🇳Kunming, China
Investigational Site Number : 1560013
🇨🇳Lanzhou, China
Investigational Site Number : 1560003
🇨🇳Shanghai, China
Investigational Site Number : 1560006
🇨🇳Suzhou, China
Investigational Site Number : 3720001
🇮🇪Crumlin, Dublin, Ireland
Investigational Site Number : 4580001
🇲🇾Johor Bahru, Malaysia
Investigational Site Number : 3920005
🇯🇵Isehara, Kanagawa, Japan
Investigational Site Number : 4580002
🇲🇾Kota Kinabalu, Malaysia
Investigational Site Number : 8040003
🇺🇦Kyiv, Ukraine
Alliance for Childhood Diseases Site Number : 8400007
🇺🇸Las Vegas, Nevada, United States
Investigational Site Number : 1580001
🇨🇳Taipei, Taiwan
Investigational Site Number : 1580005
🇨🇳Taipei, Taiwan
Investigational Site Number : 3920004
🇯🇵Saitama, Japan
Investigational Site Number : 3920008
🇯🇵Tokyo, Japan
Investigational Site Number : 0360003
🇦🇺Prahran, Victoria, Australia
Investigational Site Number : 3560007
🇮🇳Mumbai, India
Investigational Site Number : 3920002
🇯🇵Nishinomiya, Hyogo, Japan
Investigational Site Number : 1580003
🇨🇳Taipei, Taiwan
Investigational Site Number : 2080001
🇩🇰Copenhagen, Denmark
Investigational Site Number : 7920005
🇹🇷Istanbul, Turkey
Phoenix Childrens Hospital Site Number : 8400009
🇺🇸Phoenix, Arizona, United States