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
Public ClinicalTrials.gov record NCT03754790. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, 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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT03754790
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 281 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 12 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 8, 2019
- Primary completion
- Nov 4, 2026
- Completion
- Nov 4, 2026
- Last update posted
- Dec 16, 2025
2019 – 2026
United States locations
- U.S. sites
- 10
- U.S. states
- 8
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Childrens Hospital Site Number : 8400009 | Phoenix | Arizona | 85016 | — |
| Children's Hospital Los Angeles Site Number : 8400019 | Los Angeles | California | 90027 | — |
| Center for Inherited Blood Disorders (CIBD) Site Number : 8400016 | Orange | California | 92868 | — |
| Nemours Children's Clinic Site Number : 8400008 | Jacksonville | Florida | 32207 | — |
| St Joseph's Children's Hospital of Tampa Site Number : 8400002 | Tampa | Florida | 33607 | — |
| Rush University Medical Center -1725 W Harrison St Site Number : 8400001 | Chicago | Illinois | 60612-3863 | — |
| ~Massachusetts General Hospital Site Number : 8400011 | Boston | Massachusetts | 02114 | — |
| University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012 | Ann Arbor | Michigan | 48109-5000 | — |
| Alliance for Childhood Diseases Site Number : 8400007 | Las Vegas | Nevada | 89135-3011 | — |
| Childrens Hospital Medical Center of Akron Site Number : 8400006 | Akron | Ohio | 44308 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 69 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03754790, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 16, 2025 · Synced Sep 2, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03754790 live on ClinicalTrials.gov.